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Journal articles on the topic 'Personalized gene therapy'

Author: Grafiati

Published: 14 March 2023

Last updated: 31 July 2025

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1

Grinstein, Jonathan D. "Personalized Gene Therapy for All." Inside Precision Medicine 12, no. 2 (2025): 18–20. https://doi.org/10.1089/ipm.12.02.06.

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Schaly, Sabrina, Merry Ghebretatios, and Satya Prakash. "Baculoviruses in Gene Therapy and Personalized Medicine." Biologics: Targets and Therapy Volume 15 (April 2021): 115–32. http://dx.doi.org/10.2147/btt.s292692.

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Vemula, Greeshma¹ Dr. C. Bhuvaneswar Rao² Dr. Venu Gopal³. "The Impact of Artificial Intelligence in Gene Therapy." International Journal of Scientific Research and Technology 1, no. 11 (2024): 185–201. https://doi.org/10.5281/zenodo.14233536.

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Artificial intelligence (AI) is revolutionizing the field of gene therapy by enabling more precise, efficient, and personalized treatments. AI tools, particularly machine learning (ML) and deep learning (DL) algorithms, are being leveraged to analyze large genomic datasets, identify potential gene targets, and predict the outcomes of genetic modifications. These AI-driven approaches can optimize gene editing techniques like CRISPR-Cas9 by predicting off-target effects and improving editing accuracy. Moreover, AI enhances the design of gene delivery systems, aiding in the development of safer a

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Harris, Tim. "Gene and drug matrix for personalized cancer therapy." Nature Reviews Drug Discovery 9, no. 8 (2010): 660. http://dx.doi.org/10.1038/nrd3181-c1.

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Kohno, Takashi, Koji Tsuta, Katsuya Tsuchihara, Takashi Nakaoku, Kiyotaka Yoh, and Koichi Goto. "RETfusion gene: Translation to personalized lung cancer therapy." Cancer Science 104, no. 11 (2013): 1396–400. http://dx.doi.org/10.1111/cas.12275.

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Karra, Geetha, Abhiram Ch, Hanisha Tungala, Anusha Suddala, Manoj Reddy Ch, and Rama Rao Tadikonda. "Artificial Intelligence-Driven Advances in Haemophilia Gene Therapy." International Journal of Current Science Research and Review 08, no. 02 (2025): 680–87. https://doi.org/10.5281/zenodo.14824075.

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Abstract : Hemophilia is the most frequent severe genetic haemorrhagic condition. Hemophilia A and B are caused by a lack or dysfunction of the factor VIII and factor IX proteins, respectively, and are distinguished by prolonged and heavy bleeding after minor trauma or even spontaneously. Treatments for hemophilia have been extremely expensive and required the infusion of plasma clotting factors throughout one’s life. The last few years have brought major breakthroughs in gene therapy that now hold real promise for possible curative options. Artificial intelligence has the potential to t

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Kamsochukwu Ego, Obi, Ojong Naomi Agbor, and Ola-Oluwa Samuel Ayomide. "Assessing the Actuarial Implications of Gene Therapy and Personalized Medicine in Nigeria: A Case Study of Cancer Treatment." International Journal of Research and Scientific Innovation XI, no. XV (2024): 139–46. http://dx.doi.org/10.51244/ijrsi.2024.11150010p.

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This study investigates the actuarial implications of gene therapy and personalized medicine in cancer treatment in Anambra State, Nigeria. The research design was a descriptive survey, with 60 patients diagnosed with cancer from 20 general hospitals in Anambra State selected using purposive sampling. The questionnaire, titled “Questionnaire on Actuarial Implications of Gene Therapy and Personalized Medicine for Cancer Treatment” (QAIGPMCT), was structured and validated. The Cronbach Alpha Method was used to determine internal consistency and descriptive statistics of mean were used to answer

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Iacobas, Sanda, and Dumitru Andrei Iacobas. "Personalized 3-Gene Panel for Prostate Cancer Target Therapy." Current Issues in Molecular Biology 44, no. 1 (2022): 360–82. http://dx.doi.org/10.3390/cimb44010027.

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Many years and billions spent for research did not yet produce an effective answer to prostate cancer (PCa). Not only each human, but even each cancer nodule in the same tumor, has unique transcriptome topology. The differences go beyond the expression level to the expression control and networking of individual genes. The unrepeatable heterogeneous transcriptomic organization among men makes the quest for universal biomarkers and “fit-for-all” treatments unrealistic. We present a bioinformatics procedure to identify each patient’s unique triplet of PCa Gene Master Regulators (GMRs) and predic

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Barthélémy, Florian, and Nicolas Wein. "Personalized gene and cell therapy for Duchenne Muscular Dystrophy." Neuromuscular Disorders 28, no. 10 (2018): 803–24. http://dx.doi.org/10.1016/j.nmd.2018.06.009.

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Sneha, Kumari* Dr. Rakhi Kapadiya Dr. Jitendra Banweer. "Biopharmaceuticals And Gene Therapy." International Journal of Pharmaceutical Sciences 3, no. 5 (2025): 2289–98. https://doi.org/10.5281/zenodo.15409831.

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Biopharmaceuticals and gene therapy are new and powerful tools in today’s medicine. They offer precise and innovative ways to treat complex diseases. Biopharmaceuticals are made with biotechnology and include proteins for therapy, antibodies that target specific cells, and vaccines. These treatments work well with fewer side effects. Gene therapy changes or replaces faulty genes to help fight or stop diseases like cancer, genetic illnesses, and viral infections. Recent progress in methods to deliver genes, such as using viruses or other carriers, has made these treatments safer and more

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Younis, Mahmoud, and Hideyoshi Harashima. "Understanding Gene Involvement in Hepatocellular Carcinoma: Implications for Gene Therapy and Personalized Medicine." Pharmacogenomics and Personalized Medicine Volume 17 (May 2024): 193–213. http://dx.doi.org/10.2147/pgpm.s431346.

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Sahoo, Firoj Kumar. "Enalapril and the VEGFA gene: Personalized medicine in hypertension therapy." European Journal of Clinical Pharmacology 72, no. 1 (2015): 125. http://dx.doi.org/10.1007/s00228-015-1984-y.

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BERTOLOTTI, ROGER. "EDITORIAL: "AUTOLOGOUS STEM CELL GENE THERAPY: TOWARD A UNIVERSAL PLATFORM FOR PERSONALIZED THERAPY"." Gene Therapy and Regulation 03, no. 01 (2007): 1–14. http://dx.doi.org/10.1142/s1568558607000022.

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Zimbuga M., Kagambira. "Personalized Immunotherapy for Congenital Immune Defects: Precision Approaches to Immune Reconstitution." Research Output Journal of Engineering and Scientific Research 4, no. 3 (2025): 95–101. https://doi.org/10.59298/rojesr/2025/4.3.95101.

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Congenital immune defects, or primary immunodeficiency disorders (PIDs), represent a diverse group of genetically determined conditions that compromise immune function from birth. Recent advances in genomics, gene editing, and cell-based therapies have catalyzed a shift toward personalized immunotherapy—strategies that target the molecular root cause of each disorder while minimizing off-target effects. This review highlights the current landscape of precision approaches for immune reconstitution, including gene therapy, hematopoietic stem cell transplantation (HSCT), and targeted biologics. G

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Kiseleva, T. A., F. V. Valeeva, and D. R. Islamova. "Personalized therapy of type 2 diabetes mellitus." Perm Medical Journal 40, no. 5 (2023): 73–79. http://dx.doi.org/10.17816/pmj40573-79.

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Currently, the contribution of genetic factors to the development of type 2 diabetes is becoming more obvious. Despite the available nine classes of hypoglycemic drugs, only 35–40 % of patients achieve an adequate glycemic control. One the reasons may be the genetic heterogeneity of diabetes mellitus. An increasing number of studies indicates that an individual set of gene polymorphisms can determine the therapeutic response to a particular drug and cause the development of undesirable effects. The article presents an overview of a new direction in the diagnosis and treatment of diabetes melli

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Popović, Mitar, and Marina Jakšić. "Personalized medicine and pharmacogenomics." PONS - medicinski casopis 21, no. 2 (2024): 60–68. https://doi.org/10.5937/pomc21-51232.

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Personalized medicine refers to any clinical approach that emphasizes the systematic use of preventive, diagnostic, and therapeutic interventions based on genomic and family history data to enhance health outcomes. In this sense, personalized treatment is frequently referred to as genomic medicine. Personalized medicine has several health strategies that rely on the identification of molecular-genetic markers, including molecular diagnosis, prognosis, and disease monitoring; predictive genetics and preventive medicine; pharmacogenomics; targeted molecular therapy, and gene-based therapy. This

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Dugo, Ketty, Francesca Bruno, Valentina Sturiale, Desiree Brancato, Salvatore Saccone, and Concetta Federico. "Hereditary Transthyretin-Related Amyloidosis: Genetic Heterogeneity and Early Personalized Gene Therapy." Biomedicines 10, no. 10 (2022): 2394. http://dx.doi.org/10.3390/biomedicines10102394.

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Point mutations of the transthyretin (TTR) gene are related with hereditary amyloidosis (hATTR). The number of people affected by this rare disease is only partially estimated. The real impact of somatic mosaicism and other genetic factors on expressivity, complexity, progression, and transmission of the disease should be better investigated. The relevance of this rare disease is increasing and many efforts have been made to improve the time to diagnosis and to estimate the real number of cases in endemic and non-endemic areas. In this context, somatic mosaicism should be better investigated t

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M, Mavroudi, Zarogoulidis P, Porpodis K, et al. "Stem cells’ guided gene therapy of cancer: New frontier in personalized and targeted therapy." Journal of Cancer Research & Therapy 2, no. 1 (2014): 22–33. http://dx.doi.org/10.14312/2052-4994.2014-4.

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NAKAMURA, Tomonori, and Koujirou YAMAMOTO. "2. Application of Gene Analysis and TDM in Personalized Drug Therapy." Rinsho yakuri/Japanese Journal of Clinical Pharmacology and Therapeutics 44, no. 4 (2013): 361–62. http://dx.doi.org/10.3999/jscpt.44.361.

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Varin, Juliette, Clément Morival, Noémien Maillard, Oumeya Adjali, and Therese Cronin. "Risk Mitigation of Immunogenicity: A Key to Personalized Retinal Gene Therapy." International Journal of Molecular Sciences 22, no. 23 (2021): 12818. http://dx.doi.org/10.3390/ijms222312818.

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Gene therapy (GT) for ocular disorders has advanced the most among adeno-associated virus (AAV)-mediated therapies, with one product already approved in the market. The bank of retinal gene mutations carefully compiled over 30 years, the small retinal surface that does not require high clinical vector stocks, and the relatively immune-privileged environment of the eye explain such success. However, adverse effects due to AAV-delivery, though rare in the retina have led to the interruption of clinical trials. Risk mitigation, as the key to safe and efficient GT, has become the focus of ‘bedside

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Kohno, Takashi, Koji Tsuta, Katsuya Tsuchihara, Tatsuji Mizukami, Kiyotaka Yoh, and Koichi Goto. "Abstract A32: RET fusion gene: Translation to personalized lung cancer therapy." Clinical Cancer Research 20, no. 2 Supplement (2014): A32. http://dx.doi.org/10.1158/1078-0432.14aacriaslc-a32.

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Vasireddy, Vidyullatha, Jason A. Mills, Rajashekhar Gaddameedi, et al. "AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models." PLoS ONE 8, no. 5 (2013): e61396. http://dx.doi.org/10.1371/journal.pone.0061396.

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23

Abrahams, Edward. "Market access in the era of personalized cell & gene therapy." Cell and Gene Therapy Insights 5, no. 8 (2019): 971–74. http://dx.doi.org/10.18609/cgti.2019.105.

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Gambardella, Valentina, Noelia Tarazona, Juan Miguel Cejalvo, et al. "Personalized Medicine: Recent Progress in Cancer Therapy." Cancers 12, no. 4 (2020): 1009. http://dx.doi.org/10.3390/cancers12041009.

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Translational research has revolutionized how we develop new treatments for cancer patients. The change from an organ-centric concept guiding treatment choice towards deep molecular analysis, driving a personalized approach, is one of the most important advances of modern oncology. Several tools such as next generation sequencing and RNA sequencing have greatly improved the capacity to detect predictive and prognostic molecular alterations. Detection of gene mutations, amplifications, and fusions has therefore altered the history of several diseases in both a localized and metastatic setting.

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Meinl, Hanna, Marcus Zeitlhöfler, Samet Kocabey, et al. "Bifunctional Immunoactive siRNAs as an Approach to Personalized AML Therapy." Blood 122, no. 21 (2013): 5036. http://dx.doi.org/10.1182/blood.v122.21.5036.5036.

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Abstract The prognosis of acute myeloid leukemia (AML) is poor due to frequent relapse after initial remission. The development of new approaches to postremission therapy for elimination of minimal residual disease remains a major scientific and clinical challenge. We strive to combine two different innovative therapeutic concepts to develop a new specific and personalized treatment for AML. siRNAs are used to knock down either a gene that drives leukemogenesis due to genetic alterations in specific cases of AML (e.g., FLT3, NPM1) or a gene that is essential for the survival of the leukemic ce

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Szekanecz, Zoltán. "Personalized medicine in rheumatology." Orvosi Hetilap 154, no. 13 (2013): 483–96. http://dx.doi.org/10.1556/oh.2013.29580.

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In rheumatology, especially in arthritides, early diagnosis and aggressive therapy may open up new dimensions of expectations, such as improvement of pain, prevention of structural, functional damage and better quality of life. Targeted (biological) therapy has brought new horizons in rheumatology. As it is a rather expensive treatment modality, it has been urgent to develop tools suitable for the prediction of therapeutic responses. Several clinical, immunological and genetic biomarkers have been established for this purpose. Among clinical markers, male sex, younger age, lower or even higher

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Xu, Jun, Hee-Jin Lee, Jia Zeng, et al. "Extracting genetic alteration information for personalized cancer therapy from ClinicalTrials.gov." Journal of the American Medical Informatics Association 23, no. 4 (2016): 750–57. http://dx.doi.org/10.1093/jamia/ocw009.

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Abstract Objective: Clinical trials investigating drugs that target specific genetic alterations in tumors are important for promoting personalized cancer therapy. The goal of this project is to create a knowledge base of cancer treatment trials with annotations about genetic alterations from ClinicalTrials.gov. Methods: We developed a semi-automatic framework that combines advanced text-processing techniques with manual review to curate genetic alteration information in cancer trials. The framework consists of a document classification system to identify cancer treatment trials from ClinicalT

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Davies, Stella M. "Pharmacogenetics, Pharmacogenomics and Personalized Medicine: Are We There Yet?" Hematology 2006, no. 1 (2006): 111–17. http://dx.doi.org/10.1182/asheducation-2006.1.111.

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Abstract The genetic basis of a differential response to drugs has been understood for a limited number of agents for over 30 years. This knowledge has generated hope that the individual basis for response to a wide range of drugs would be quickly known, and individualized drug selection and dosing would be possible for many or all disorders. Understanding the variable response to drugs seems particularly pressing in the field of oncology, in which the stakes are high (failure to cure cancer usually leads to death), drugs commonly have a narrow therapeutic index, and toxicities can be severe (

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Jeleriu, Roxana Maria, Roxana-Karin Hajaj, Iuliana-Anamaria Trăilă, Mihaela Zaharie, and Maria Puiu. "Theoretical Models and Simulations of Gene Delivery with Polyurethane: The Importance of Polyurethane as a Vector in Personalized Therapy." Biomedicines 13, no. 3 (2025): 692. https://doi.org/10.3390/biomedicines13030692.

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Background/Objectives: Advancements in personalized medicine have revolutionized drug delivery, enabling tailored treatments based on genetic and molecular profiles. Non-viral vectors, such as polyurethane (PU)-based systems, offer promising alternatives for gene therapy. This study develops mathematical models to analyze PU degradation, DNA/RNA release kinetics, and cellular interactions, optimizing their application in personalized therapy. Methods: This theoretical study utilized mathematical modeling and numerical simulations to analyze PU-based gene delivery, focusing on diffusion, degrad

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Hubert, E. Blum. "Advances in personalized gastroenterology and hepatology 2016." Russian Journal of Gastroenterology, Hepatology, Coloproctology 26, no. 3 (2016): 4–10. http://dx.doi.org/10.22416/1382-4376-2016-26-3-4-10.

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Molecular and cell biology have resulted in major advances in our understanding of disease pathogenesis as well as in novel strategies for the diagnosis, therapy and prevention of human diseases. Based on modern molecular, genetic, epigenetic microbiologic and biochemical analyses it is, on the one hand, possible to identify disease-related point mutations and single nucleotide polymorphisms in the context of genomewide association analyses (GWAS). On the other hand, using high throughput array and other technologies, it is possible to simultaneously analyze thousands of genes (DNA) or gene pr

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Chetina, E. V., and G. A. Markova. "Upcoming value of gene expression analysis in rheumatology." Biomeditsinskaya Khimiya 64, no. 3 (2018): 221–32. http://dx.doi.org/10.18097/pbmc20186403221.

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Rheumatoid arthritis (RA) is a chronic inflammatory disease of unknown etiology, which involves disturbance in immune system signaling pathway functions, damage of other tissues, pain and joint destruction. Modern treatment attempts to improve pathophysiological and biochemical mechanisms damaged by the disease. However, due to the RA patient heterogeneity personalized approach to treatment is required; the choice of personalized treatment is complicated by the variability of patient's response to treatment. Gene expression analysis might serve a tool for the disease control and therapy person

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Obutuzi G., Nakaziya. "Congenital Defects in the Immune System: A Pathway to Personalized Immunotherapy." NEWPORT INTERNATIONAL JOURNAL OF RESEARCH IN MEDICAL SCIENCES 6, no. 2 (2025): 11–14. https://doi.org/10.59298/nijrms/2025/6.2.1114.

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Congenital defects in the immune system, collectively known as primary immunodeficiency disorders (PIDs), present significant clinical challenges due to their impact on host defense mechanisms. These disorders result from genetic mutations affecting various components of the innate and adaptive immune systems, leading to recurrent infections, autoimmunity, and increased susceptibility to malignancies. Recent advancements in immunogenetics and precision medicine have paved the way for personalized immunotherapy approaches tailored to individual immune defects. This review explores the pathophys

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Khan, Sikandar Hayat. "Type-2 Diabetes and Gene Therapy: The Promise of CRISPR Gene Therapy in type-2 Diabetes Mellitus." Journal Of Obesity Management 1, no. 3 (2019): 1–5. http://dx.doi.org/10.14302/issn.2574-450x.jom-19-3001.

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Gene therapy has entered a new era with the dawn of CRISPR/Cas9 technology which though were always available in nature but rediscovered to tame into a real-tlife genome editing tool. With the modernization upsurge and changes in ways the “homo sapiens” survived on this planet from hunger to current era of exuberance has led to multiple metabolic issues like type-2 diabetes. Notwithstanding the rapid emergence of medication to suppress the hyperglycemia and insulin resistance associated with this menace, need has definitely emerge to find more personalized and curative dimensions to therapeuti

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Udayaraja, G. K., and I. Arnold Emerson. "Oncogenomics and CYP450 Implications in Personalized Cancer Therapy." Current Pharmacogenomics and Personalized Medicine 17, no. 2 (2020): 104–13. http://dx.doi.org/10.2174/1875692117999200517122652.

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Background: The Human Genome Project has unleashed the power of genomics in clinical practice as a choice of individualized therapy, particularly in cancer treatment. Pharmacogenomics is an interdisciplinary field of genomics that deals with drug response, based on individual genetic makeup. Objective: The main genetic events associated with carcinogenesis activate oncogenes or inactivate tumor-suppressor genes. Therefore, drugs should be specific to inactivate or regulate these mutant genes and their protein products for effective cancer treatment. In this review, we summarize how polymedicat

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Gui, Yifeng. "Personalized neoantigen-based therapeutic cancer vaccines." Theoretical and Natural Science 40, no. 1 (2024): 85–89. http://dx.doi.org/10.54254/2753-8818/40/20241222.

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Abstract. Tumor neoantigens are new unknown proteins or peptides encoded by somatic gene mutations, also known as tumor-specific antigens (TSAs). They are located on the surface of tumor cells and are only expressed in tumor cells. Cancer vaccines targeting neoantigens can not only effectively induce immune response, but also have the advantages of high safety and fewer side effects, so it has gradually become a research hotspot in tumor therapy. During the current clinical trials, neoantigen-based personalised tumour therapeutic vaccines have shown excellent experimental results and have also

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Cai, Xiaochen, and Xiaoming Zhu. "Anorectal melanoma and gene analysis of personalized adjuvant therapy: a case report." Annals of Palliative Medicine 10, no. 10 (2021): 11216–20. http://dx.doi.org/10.21037/apm-21-2240.

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Vasireddy, Vidyullatha, Jason A. Mills, Rajashekhar Gaddameedi, et al. "Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models." PLOS ONE 10, no. 6 (2015): e0129982. http://dx.doi.org/10.1371/journal.pone.0129982.

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Sureda, Manuel, Ramon Gonzalez Manzano, Elena Ma Martínez, et al. "Personalized gene expression profiling-guided (MAGE) therapy in resistant sarcomas. Preliminary data." Journal of Clinical Oncology 34, no. 15_suppl (2016): e14028-e14028. http://dx.doi.org/10.1200/jco.2016.34.15_suppl.e14028.

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Patsali, Petros, Claudio Mussolino, Constantinos Loucari, et al. "692. Genome Editing for Personalized Gene Therapy of IVSI-110 Beta-Thalassemia." Molecular Therapy 24 (May 2016): S274. http://dx.doi.org/10.1016/s1525-0016(16)33500-6.

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Zhang, Qun, Lei Cheng, Jing Hu, et al. "SLAMF8, a novel biomarker for personalized immune checkpoint blockade therapy in gastrointestinal cancer." Journal of Clinical Oncology 37, no. 15_suppl (2019): e14078-e14078. http://dx.doi.org/10.1200/jco.2019.37.15_suppl.e14078.

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e14078 Background: Immune checkpoint inhibitors have brought great breakthroughs in cancer therapy. Activated immune response is known to be the prerequisite for exerting immunotherapy efficacy. Epstein-Barr virus (EBV) infection is associated with longer survival in gastric cancer (GC) patients due to enhanced anti-tumor immune response, and therefore it was reportedly played an important role in modulating immune checkpoint blockade therapy efficacy. However, molecular dimensions underlying the good response to immune checkpoint inhibitors in presence of EBV infection are still unclear. The

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Dr., Swapna Moparthi*, Priyadharshini S., Sowjanya Kondru Dr., et al. "A REVIEW ON THE THERAPEUTIC POTENTIAL OF GENE THERAPY IN NEUROLOGICAL DISORDERS." World Journal of Pharmaceutical Science and Research 3, no. 4 (2024): 212–24. https://doi.org/10.5281/zenodo.13320814.

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Gene therapy is a novel way to treating neurological illnesses, potentially curing some of the most difficult and incurable conditions at this time. This review examines the therapeutic potential of gene therapy for a range of neurological illnesses, with an emphasis on the mechanisms underlying these effects, such as cell-based therapies, gene silencing, gene editing, gene insertion, and gene control. Gene therapy offers intriguing pathways to address the underlying causes of neurological illnesses by repairing genetic defects, lowering toxic protein levels, improving neuroprotection, restori

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Morroni, Fabiana, and Antonella Caccamo. "Advances and Challenges in Gene Therapy for Alzheimer’s Disease." Journal of Alzheimer's Disease 101, s1 (2024): S417—S431. http://dx.doi.org/10.3233/jad-230783.

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Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by cognitive decline, memory loss, and behavioral impairments. Despite extensive research efforts, effective treatment options for AD remain limited. Recently, gene therapy has emerged as a promising avenue for targeted intervention in the pathogenesis of AD. This review will provide an overview of clinical and preclinical studies where gene therapy techniques have been utilized in the context of AD, highlighting their potential as novel therapeutic strategies. While challenges remain, ongoing research and techn

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Iacobas, Sanda, Nneka Ede, and Dumitru A. Iacobas. "The Gene Master Regulators (GMR) Approach Provides Legitimate Targets for Personalized, Time-Sensitive Cancer Gene Therapy." Genes 10, no. 8 (2019): 560. http://dx.doi.org/10.3390/genes10080560.

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The dynamic and never exactly repeatable tumor transcriptomic profile of people affected by the same form of cancer requires a personalized and time-sensitive approach of the gene therapy. The Gene Master Regulators (GMRs) were defined as genes whose highly controlled expression by the homeostatic mechanisms commands the cell phenotype by modulating major functional pathways through expression correlation with their genes. The Gene Commanding Height (GCH), a measure that combines the expression control and expression correlation with all other genes, is used to establish the gene hierarchy in

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Suwanmanee, Thipparat, Martin T. Ferris, Peirong Hu, et al. "Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery." Molecular Therapy - Methods & Clinical Development 5 (June 2017): 83–92. http://dx.doi.org/10.1016/j.omtm.2017.03.009.

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Abdulsalam, Mustapha, Fatima Umar Hamza, Fatima Abubakar Saddeeq, Hafsa Hamisu Ibrahim, Hafsa Ahmad Isa Dutse, and Aisha Mustapha Falaki. "Unveiling the Molecular Symphony: Exploring Mechanisms, Diversity and Applications of Restriction Enzymes in Biology." International Journal of Applied and Scientific Research 2, no. 3 (2024): 325–42. http://dx.doi.org/10.59890/ijasr.v2i3.1554.

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This research delves into the multifaceted role of restriction enzymes in molecular biology, examining their historical evolution, intricate mechanisms, and diverse applications. It aims to comprehensively understand their versatility and integration with modern technologies like CRISPR-Cas9, particularly in personalized medicine and gene therapy. Addressing a research gap in the synergism of restriction enzymes with CRISPR-Cas9 and their role in epigenetic modifications, qualitative methods critically assess existing literature and propose future research models. Findings highlight the potent

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Kirsanov, Kirill I., Ekaterina A. Lesovaya, Timur I. Fetisov, Beniamin Yu Bokhyan, Gennady A. Belitsky, and Marianna G. Yakubovskaya. "Current Approaches for Personalized Therapy of Soft Tissue Sarcomas." Sarcoma 2020 (April 1, 2020): 1–15. http://dx.doi.org/10.1155/2020/6716742.

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Soft tissue sarcomas (STS) are a highly heterogeneous group of cancers of mesenchymal origin with diverse morphologies and clinical behaviors. While surgical resection is the standard treatment for primary STS, advanced and metastatic STS patients are not eligible for surgery. Systemic treatments, including standard chemotherapy and newer chemical agents, still play the most relevant role in the management of the disease. Discovery of specific genetic alterations in distinct STS subtypes allowed better understanding of mechanisms driving their pathogenesis and treatment optimization. This revi

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Lin, Luqi, Zhengrong Cui, Sihao Wang, Yizhi Chen, and Yanqi Zong. "AI-driven Protein Engineering for DNA Sequence Modification." Journal of Theory and Practice of Engineering Science 4, no. 03 (2024): 183–90. http://dx.doi.org/10.53469/jtpes.2024.04(03).17.

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The integration of artificial intelligence (AI) with gene editing technologies like CRISPR-Cas9 holds immense promise for advancing biomedical research and personalized medicine. This article highlights the crucial role of AI in predicting and minimizing off-target effects, thereby enhancing the precision and efficiency of gene editing. Researchers have developed algorithms like BE-DICT to accurately predict base editing outcomes, showcasing the potential of AI-driven strategies in optimizing gene editing processes. By combining AI with bioengineering, this interdisciplinary approach aims to a

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Birimberg-Schwartz, Liron, Wan Ip, Claire Bartlett, et al. "Validating organoid-derived human intestinal monolayers for personalized therapy in cystic fibrosis." Life Science Alliance 6, no. 6 (2023): e202201857. http://dx.doi.org/10.26508/lsa.202201857.

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Highly effective drugs modulating the defective protein encoded by the CFTR gene have revolutionized cystic fibrosis (CF) therapy. Preclinical drug-testing on human nasal epithelial (HNE) cell cultures and 3-dimensional human intestinal organoids (3D HIO) are used to address patient-specific variation in drug response and to optimize individual treatment for people with CF. This study is the first to report comparable CFTR functional responses to CFTR modulator treatment among patients with different classes of CFTR gene variants using the three methods of 2D HIO, 3D HIO, and HNE. Furthermore,

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Raghuram, Anjali, Aspinder Singh, Daniel K. Chang, Mervin Nunez, Edward M. Reece, and Brent E. Schultz. "The Evolving Landscape of Gene Therapy in Plastic Surgery." Seminars in Plastic Surgery 33, no. 03 (2019): 167–72. http://dx.doi.org/10.1055/s-0039-1693131.

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AbstractWith the rapid rise of personalized genomic sequencing and clustered regularly interspaced short palindromic repeat (CRISPR) technology, previous gaps in gene therapy are beginning to be bridged, paving the way for increasing clinical applicability. This article aims to provide an overview of the fundamentals of gene therapy and discuss future potential interventions relevant to plastic surgeons. These interventions include enhancing tissue regeneration and healing, as well as modifying disease processes in congenital anomalies. Though clinical applications are still on the horizon, a

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Terzic, M., M. Jakimovska, S. Fustik, T. Jakovska, E. Sukarova-Stefanovska, and D. Plaseska-Karanfilska. "Cystic fibrosis mutation spectrum in north macedonia: A step toward personalized therapy." Balkan Journal of Medical Genetics 22, no. 1 (2019): 35–40. http://dx.doi.org/10.2478/bjmg-2019-0009.

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AbstractThe most prevalent "rare" disease worldwide, cystic fibrosis (CF), is an autosomal recessive multisystem disease, caused by mutations in the CFTR gene. The knowledge of CFTR mutations present in certain population is important for designing a simple, fast and cost-effective genetic testing approach, also for better management of CF patients, including the administration of novel targeted therapies. Here, we present genetic results of 158 unrelated CF patients from the National CF Registry of the Republic of North Macedonia. Initially, patients were screened for the 11 most common CF mu

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