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Relevant bibliographies by topics / CRISPR, Gene editing, Parkinson, Gene therapy

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Journal articles

Academic literature on the topic 'CRISPR, Gene editing, Parkinson, Gene therapy'

Author: Grafiati

Published: 14 March 2023

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Journal articles on the topic "CRISPR, Gene editing, Parkinson, Gene therapy"

1

Chung, Sun-Ku, and Seo-Young Lee. "Advances in Gene Therapy Techniques to Treat LRRK2 Gene Mutation." Biomolecules 12, no. 12 (2022): 1814. http://dx.doi.org/10.3390/biom12121814.

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Leucine-rich repeat kinase 2 (LRRK2) gene mutation is an autosomal dominant mutation associated with Parkinson’s disease (PD). Among LRRK2 gene mutations, the LRRK2 G2019S mutation is frequently involved in PD onset. Currently, diverse gene correction tools such as zinc finger nucleases (ZFNs), helper-dependent adenoviral vector (HDAdV), the bacterial artificial chromosome-based homologous recombination (BAC-based HR) system, and CRISPR/Cas9-homology-directed repair (HDR) or adenine base editor (ABE) are used in genome editing. Gene correction of the LRRK2 G2019S mutation has been applied when

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2

Rahman, Mujeeb ur, Muhammad Bilal, Junaid Ali Shah, Ajeet Kaushik, Pierre-Louis Teissedre, and Małgorzata Kujawska. "CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease." Pharmaceutics 14, no. 6 (2022): 1252. http://dx.doi.org/10.3390/pharmaceutics14061252.

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Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques h

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3

De Plano, Laura M., Giovanna Calabrese, Sabrina Conoci, Salvatore P. P. Guglielmino, Salvatore Oddo, and Antonella Caccamo. "Applications of CRISPR-Cas9 in Alzheimer’s Disease and Related Disorders." International Journal of Molecular Sciences 23, no. 15 (2022): 8714. http://dx.doi.org/10.3390/ijms23158714.

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Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and Huntington’s disease represent some of the most prevalent neurodegenerative disorders afflicting millions of people worldwide. Unfortunately, there is a lack of efficacious treatments to cure or stop the progression of these disorders. While the causes of such a lack of therapies can be attributed to various reasons, the disappointing results of recent clinical trials suggest the need for novel and innovative approaches. Since its discovery, there has been a growing excitement around the potential for CRISPR-Cas9 medi

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4

Katzmann, Julius L., Arjen J. Cupido, and Ulrich Laufs. "Gene Therapy Targeting PCSK9." Metabolites 12, no. 1 (2022): 70. http://dx.doi.org/10.3390/metabo12010070.

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The last decades of research in cardiovascular prevention have been characterized by successful bench-to-bedside developments for the treatment of low-density lipoprotein (LDL) hypercholesterolemia. Recent examples include the inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9) with monoclonal antibodies, small interfering RNA and antisense RNA drugs. The cumulative effects of LDL cholesterol on atherosclerosis make early, potent, and long-term reductions in LDL cholesterol desirable—ideally without the need of regular intake or application of medication and importantly, withou

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5

Tang, Xuanting. "CRISPR/Cas9-based genome engineering in HIV gene therapy." E3S Web of Conferences 233 (2021): 02004. http://dx.doi.org/10.1051/e3sconf/202123302004.

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In recent years, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated nuclease (Cas) technology has become the most heated genome editing technique. Comparing to earlier genetic engineering methods, the CRISPR/Cas system is more advantageous due to its simple convenient design, high efficiency, cost-effectiveness, and the ability to perform multi-sites editing simultaneously. As the most effective gene editing tool, it utilizes a simple short RNA-guided mechanism to direct Cas-mediated DNA cleavage at the target genome locus and exploits the endogenous DNA r

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6

S., Manasa M. "CRISPR-Cas9 gene editing technology in human gene therapy: the new realm of medicine." International Journal of Advances in Medicine 9, no. 4 (2022): 513. http://dx.doi.org/10.18203/2349-3933.ijam20220796.

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Gene therapy has a huge clinical relevance in the present therapeutic world and is one of the many research fields of biology which received many benefits from the recent advancements of modern clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing technology. Researchers are on the way to make significant changes in the ways of treating genetic abnormalities. An increase in the number of approved clinical trials of CRISPR based gene therapy shows we are not too far from eliminating deadly diseases such as acquired immunodeficiency syndrome (AIDS), cancer and many

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7

Preece, Roland, and Christos Georgiadis. "Emerging CRISPR/Cas9 applications for T-cell gene editing." Emerging Topics in Life Sciences 3, no. 3 (2019): 261–75. http://dx.doi.org/10.1042/etls20180144.

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Abstract Gene editing tools are being rapidly developed, accelerating many areas of cell and gene therapy research. Each successive gene editing technology promises increased efficacy, improved specificity, reduced manufacturing cost and design complexity; all of which are currently epitomised by the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas9) platform. Since its conceptualisation, CRISPR-based gene editing has been applied to existing methodologies and has further allowed the exploration of novel avenues of research. Implementation o

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8

Liu, Wenlou, Chunsheng Yang, Yanqun Liu, and Guan Jiang. "CRISPR/Cas9 System and its Research Progress in Gene Therapy." Anti-Cancer Agents in Medicinal Chemistry 19, no. 16 (2020): 1912–19. http://dx.doi.org/10.2174/1871520619666191014103711.

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Genome editing refers to changing the genome sequence of an organism by knockout, insertion, and site mutation, resulting in changes in the genetic information of the organism. The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein-9 nuclease (Cas9) system is a genome editing technique developed by the acquired immune system in the microbes, such as bacteria and archaebacteria, which targets and edits genome sequences according to the principle of complementary base pairing. This technique can be used to edit endogenous genomic DNA sequences in organi

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9

Salsman, Jayme, and Graham Dellaire. "Precision genome editing in the CRISPR era." Biochemistry and Cell Biology 95, no. 2 (2017): 187–201. http://dx.doi.org/10.1139/bcb-2016-0137.

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With the introduction of precision genome editing using CRISPR–Cas9 technology, we have entered a new era of genetic engineering and gene therapy. With RNA-guided endonucleases, such as Cas9, it is possible to engineer DNA double strand breaks (DSB) at specific genomic loci. DSB repair by the error-prone non-homologous end-joining (NHEJ) pathway can disrupt a target gene by generating insertions and deletions. Alternatively, Cas9-mediated DSBs can be repaired by homology-directed repair (HDR) using an homologous DNA repair template, thus allowing precise gene editing by incorporating genetic c

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10

Kanu, Gayathri A., Javad B. M. Parambath, Raed O. Abu Odeh, and Ahmed A. Mohamed. "Gold Nanoparticle-Mediated Gene Therapy." Cancers 14, no. 21 (2022): 5366. http://dx.doi.org/10.3390/cancers14215366.

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Gold nanoparticles (AuNPs) have gained increasing attention as novel drug-delivery nanostructures for the treatment of cancers, infections, inflammations, and other diseases and disorders. They are versatile in design, synthesis, modification, and functionalization. This has many advantages in terms of gene editing and gene silencing, and their application in genetic illnesses. The development of several techniques such as CRISPR/Cas9, TALEN, and ZFNs has raised hopes for the treatment of genetic abnormalities, although more focused experimentation is still needed. AuNPs, however, have been mu

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