Academic literature on the topic 'Genetic Therapy'

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Journal articles on the topic "Genetic Therapy"

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Flotte, Terence R., and Thomas W. Ferkol. "GENETIC THERAPY." Pediatric Clinics of North America 44, no. 1 (February 1997): 153–78. http://dx.doi.org/10.1016/s0031-3955(05)70468-5.

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del Vecchio, F., A. Filareto, P. Spitalieri, F. Sangiuolo, and G. Novelli. "Cellular Genetic Therapy." Transplantation Proceedings 37, no. 6 (July 2005): 2657–61. http://dx.doi.org/10.1016/j.transproceed.2005.06.037.

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Harris, Jonathan, and Karol Sikora. "Human genetic therapy." Molecular Aspects of Medicine 14, no. 6 (January 1993): 451–543. http://dx.doi.org/10.1016/0098-2997(93)90021-5.

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Roth, Theodore L., and Alexander Marson. "Genetic Disease and Therapy." Annual Review of Pathology: Mechanisms of Disease 16, no. 1 (January 24, 2021): 145–66. http://dx.doi.org/10.1146/annurev-pathmechdis-012419-032626.

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Genetic diseases cause numerous complex and intractable pathologies. DNA sequences encoding each human's complexity and many disease risks are contained in the mitochondrial genome, nuclear genome, and microbial metagenome. Diagnosis of these diseases has unified around applications of next-generation DNA sequencing. However, translating specific genetic diagnoses into targeted genetic therapies remains a central goal. To date, genetic therapies have fallen into three broad categories: bulk replacement of affected genetic compartments with a new exogenous genome, nontargeted addition of exogenous genetic material to compensate for genetic errors, and most recently, direct correction of causative genetic alterations using gene editing. Generalized methods of diagnosis, therapy, and reagent delivery into each genetic compartment will accelerate the next generations of curative genetic therapies. We discuss the structure and variability of the mitochondrial, nuclear, and microbial metagenomic compartments, as well as the historical development and current practice of genetic diagnostics and gene therapies targeting each compartment.
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Emery, A. "Therapy for Genetic Disease." Journal of Medical Genetics 29, no. 2 (February 1, 1992): 142–43. http://dx.doi.org/10.1136/jmg.29.2.142-a.

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Martin, LA, R. Vile, NR Lemoine, K. Sikora, and HS Pandha. "Genetic prodrug activation therapy." Lancet 350, no. 9094 (December 1997): 1793–94. http://dx.doi.org/10.1016/s0140-6736(05)63633-1.

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Rigg, Anne, and Karol Sikora. "Genetic prodrug activation therapy." Molecular Medicine Today 3, no. 8 (August 1997): 359–66. http://dx.doi.org/10.1016/s1357-4310(97)01082-4.

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Miller, P. "Therapy for genetic diseases." Biochemical Education 19, no. 4 (October 1991): 220. http://dx.doi.org/10.1016/0307-4412(91)90112-l.

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Hirschhorn, Rochelle. "Therapy of Genetic Disorders." New England Journal of Medicine 316, no. 10 (March 5, 1987): 623–24. http://dx.doi.org/10.1056/nejm198703053161011.

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Rizzo, Robert F. "Genetic testing and therapy." International Journal of Social Economics 26, no. 1/2/3 (January 1999): 109–33. http://dx.doi.org/10.1108/03068299910229523.

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Dissertations / Theses on the topic "Genetic Therapy"

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Aryamvally, Anjali. "Mitochondrial Replacement Therapy: Genetic Counselors’ Experiences, Knowledge and Opinions." University of Cincinnati / OhioLINK, 2020. http://rave.ohiolink.edu/etdc/view?acc_num=ucin1583998248123854.

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Blechacz, Boris Roman Alexander. "Genetic approaches to the therapy of hepatocellular carcinoma." Thesis, Open University, 2009. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.505358.

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Hepatocellular carcinoma (HCC) is a devastating malignancy originating from hepatocytes. There is an urgent need for novel therapeutic approaches. Currently explored gene therapy systems have not yet achieved significant survival benefits. The aim of this thesis was the development and evaluation of novel genetic approaches to this malignancy.
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Ganly, Ian. "E1B attenuated adenoviruses in genetic therapy for cancer." Thesis, University of Glasgow, 1998. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.266588.

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Wood, David Rowe Ding Jiahuan. "Design, optimization, and evaluation of conditionally active gene therapy vectors." Waco, Tex. : Baylor University, 2008. http://hdl.handle.net/2104/5153.

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Choudhury, Sourav Roy. "Developing an Adeno-Associated Viral Vector (AAV) Toolbox for CNS Gene Therapy: A Dissertation." eScholarship@UMMS, 2016. https://escholarship.umassmed.edu/gsbs_diss/809.

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Neurological disorders – disorders of the brain, spine and associated nerves – are a leading contributor to global disease burden with a sizable economic cost. Adeno-associated viral (AAV) vectors have emerged as an effective platform for CNS gene therapy and have shown early promise in clinical trials. These trials involve direct infusion into brain parenchyma, an approach that may be suboptimal for treatment of neurodegenerative disorders, which often involve more than a single structure in the CNS. However, overall neuronal transduction efficiency of vectors derived from naturally occurring AAV capsids after systemic administration is relatively low. We have developed novel capsids AAV-AS and AAV-B1 that lead to widespread gene delivery throughout the brain and spinal cord, particularly to neuronal populations. Both transduce the adult mouse brain >10-fold more efficiently than the clinical gold standard AAV9 upon intravascular infusion, with gene transfer to multiple neuronal sub-populations. These vectors are also capable of neuronal transduction in a normal cat. We have demonstrated the efficacy of AAV-AS in the context of Huntington's disease by knocking down huntingtin mRNA 33-50% after a single intravenous injection, which is better than what can be achieved by AAV9 at the particular dose. AAVB1 additionally transduces muscle, beta cells, pulmonary alveoli and retinal vasculature at high efficiency, and has reduced sensitivity to neutralizing antibodies in human sera. Generation of this vector toolbox represents a major step towards gaining genetic access to the entire CNS, and provides a platform to develop new gene therapies for neurodegenerative disorders.
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Choudhury, Sourav Roy. "Developing an Adeno-Associated Viral Vector (AAV) Toolbox for CNS Gene Therapy: A Dissertation." eScholarship@UMMS, 2001. http://escholarship.umassmed.edu/gsbs_diss/809.

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Neurological disorders – disorders of the brain, spine and associated nerves – are a leading contributor to global disease burden with a sizable economic cost. Adeno-associated viral (AAV) vectors have emerged as an effective platform for CNS gene therapy and have shown early promise in clinical trials. These trials involve direct infusion into brain parenchyma, an approach that may be suboptimal for treatment of neurodegenerative disorders, which often involve more than a single structure in the CNS. However, overall neuronal transduction efficiency of vectors derived from naturally occurring AAV capsids after systemic administration is relatively low. We have developed novel capsids AAV-AS and AAV-B1 that lead to widespread gene delivery throughout the brain and spinal cord, particularly to neuronal populations. Both transduce the adult mouse brain >10-fold more efficiently than the clinical gold standard AAV9 upon intravascular infusion, with gene transfer to multiple neuronal sub-populations. These vectors are also capable of neuronal transduction in a normal cat. We have demonstrated the efficacy of AAV-AS in the context of Huntington's disease by knocking down huntingtin mRNA 33-50% after a single intravenous injection, which is better than what can be achieved by AAV9 at the particular dose. AAVB1 additionally transduces muscle, beta cells, pulmonary alveoli and retinal vasculature at high efficiency, and has reduced sensitivity to neutralizing antibodies in human sera. Generation of this vector toolbox represents a major step towards gaining genetic access to the entire CNS, and provides a platform to develop new gene therapies for neurodegenerative disorders.
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Massimo, Gianmichele <1985&gt. "Hypertension, hypercholesterolemia, hyperaldosteronism: a genetic perspective for personalized therapy." Doctoral thesis, Alma Mater Studiorum - Università di Bologna, 2014. http://amsdottorato.unibo.it/6473/1/PhD_Thesis_Gianmichele_Massimo.pdf.

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Essential, primary, or idiopathic hypertension is defined as high BP in which secondary causes such as renovascular disease, renal failure, pheochromocytoma, hyperaldosteronism, or other causes of secondary hypertension are not present. Essential hypertension accounts for 80-90% of all cases of hypertension; it is a heterogeneous disorder, with different patients having different causal factors that may lead to high BP. Life-style, diet, race, physical activity, smoke, cultural level, environmental factors, age, sex and genetic characteristics play a key role in the increasing risk. Conversely to the essential hypertension, secondary hypertension is often associated with the presence of other pathological conditions such as dyslipidaemia, hypercholesterolemia, diabetes mellitus, obesity and primary aldosteronism. Amongst them, primary aldosteronism represents one of the most common cause of secondary hypertension, with a prevalence of 5-15% depending on the severity of blood pressure. Besides high blood pressure values, a principal feature of primary aldosteronism is the hypersecretion of mineralcorticoid hormone, aldosterone, in a manner that is fairly autonomous of the renin-angiotensin system. Primary aldosteronism is a heterogeneous pathology that may be divided essentially in two groups, idiopathic and familial form. Despite all this knowledge, there are so many hypertensive cases that cannot be explained. These individuals apparently seem to be healthy, but they have a great risk to develop CVD. The lack of known risk factors makes difficult their classification in a scale of risk. Over the last three decades a good help has been given by the pharmacogenetics/pharmacogenomics, a new area of the traditional pharmacology that try to explain and find correlations between genetic variation, (rare variations, SNPs, mutations), and the risk to develop a particular disease.
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Massimo, Gianmichele <1985&gt. "Hypertension, hypercholesterolemia, hyperaldosteronism: a genetic perspective for personalized therapy." Doctoral thesis, Alma Mater Studiorum - Università di Bologna, 2014. http://amsdottorato.unibo.it/6473/.

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Essential, primary, or idiopathic hypertension is defined as high BP in which secondary causes such as renovascular disease, renal failure, pheochromocytoma, hyperaldosteronism, or other causes of secondary hypertension are not present. Essential hypertension accounts for 80-90% of all cases of hypertension; it is a heterogeneous disorder, with different patients having different causal factors that may lead to high BP. Life-style, diet, race, physical activity, smoke, cultural level, environmental factors, age, sex and genetic characteristics play a key role in the increasing risk. Conversely to the essential hypertension, secondary hypertension is often associated with the presence of other pathological conditions such as dyslipidaemia, hypercholesterolemia, diabetes mellitus, obesity and primary aldosteronism. Amongst them, primary aldosteronism represents one of the most common cause of secondary hypertension, with a prevalence of 5-15% depending on the severity of blood pressure. Besides high blood pressure values, a principal feature of primary aldosteronism is the hypersecretion of mineralcorticoid hormone, aldosterone, in a manner that is fairly autonomous of the renin-angiotensin system. Primary aldosteronism is a heterogeneous pathology that may be divided essentially in two groups, idiopathic and familial form. Despite all this knowledge, there are so many hypertensive cases that cannot be explained. These individuals apparently seem to be healthy, but they have a great risk to develop CVD. The lack of known risk factors makes difficult their classification in a scale of risk. Over the last three decades a good help has been given by the pharmacogenetics/pharmacogenomics, a new area of the traditional pharmacology that try to explain and find correlations between genetic variation, (rare variations, SNPs, mutations), and the risk to develop a particular disease.
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Holder, Kristina Kichler. "Dynamics of adaptive evolution in two experimental viral systems." Access restricted to users with UT Austin EID Full text (PDF) from UMI/Dissertation Abstracts International, 2001. http://wwwlib.umi.com/cr/utexas/fullcit?p3037499.

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Fuller, Maria. "A gene transfer system derived from human immunodeficiency virus type 1 (HIV-1)." Title page, table of contents, list of abbreviations and epitome only, 2001. http://web4.library.adelaide.edu.au/theses/09PH/09phf9669.pdf.

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Books on the topic "Genetic Therapy"

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David, Curiel, and Douglas Joanne T, eds. Adenoviral vectors for gene therapy. Amsterdam: Academic Press, 2002.

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1935-, Friedmann Theodore, ed. Therapy for genetic disease. Oxford: Oxford University Press, 1991.

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Dr, Cooper David N., and Lemoine Nicholas R, eds. Gene therapy. Oxford, UK: Bios Scientific Publishers, 1996.

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H, Vos Jean-Michel, ed. Viruses in human gene therapy. Durham, N.C: Carolina Academic Press, 1995.

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Vines, Gail. Diving into the gene pool: A user's guide to the new genetic medicine. London: Medical Research Council ; Wellcome Trust, 1995.

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K, Hunt Kelly, Vorburger Stephan A, and Swisher Stephen G, eds. Gene therapy for cancer. Totowa, N.J: Humana Press, 2007.

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1935-, Friedmann Theodore, ed. The development of human gene therapy. Cold Spring Harbor, NY: Cold Spring Harbor Laboratory Press, 1999.

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1936-, Anderson W. French, and National Institutes of Health (U.S.). Recombinant DNA Advisory Committee. Human Gene Therapy Subcommittee, eds. Human gene therapy: Preclinical data document. Bethesda, Md: The Institutes, 1987.

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1958-, Xanthopoulos Kleanthis, ed. Gene therapy. Berlin: Springer, 1998.

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Cady, Blake. Cancer gene therapy. Philadelphia: W.B. Saunders, 1998.

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Book chapters on the topic "Genetic Therapy"

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Cornetta, Kenneth. "Gene Therapy." In Molecular Genetic Pathology, 717–29. Totowa, NJ: Humana Press, 2008. http://dx.doi.org/10.1007/978-1-59745-405-6_29.

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Moseley, A. B., and C. T. Caskey. "Prospects for Human Gene Therapy." In Genetic Engineering, 213–23. Boston, MA: Springer US, 1993. http://dx.doi.org/10.1007/978-1-4899-1666-2_10.

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Kmiec, Eric B., Allyson Cole-Strauss, Michael C. Rice, and Pamela Havre. "Genetic Correction for Gene Therapy." In Gene Therapy, 111–18. Berlin, Heidelberg: Springer Berlin Heidelberg, 1998. http://dx.doi.org/10.1007/978-3-642-72160-1_12.

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Farmer, Diana L., Hammin Lee, Elizabeth Gress, Aubrey Milunsky, and Michael R. Harrison. "Fetal Therapy." In Genetic Disorders and the Fetus, 1001–19. Oxford, UK: Wiley-Blackwell, 2010. http://dx.doi.org/10.1002/9781444314342.ch31.

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Verp, Marion S. "Genetic Counseling." In Principles of Medical Therapy in Pregnancy, 1217–22. Boston, MA: Springer US, 1985. http://dx.doi.org/10.1007/978-1-4613-2415-7_189.

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Wynshaw-Boris, Anthony, Carrolee Barlow, Amy Chen, Michael Gambello, Lisa Garrett, Theresa Hernandez, Shinji Hirotsune, et al. "Modeling Genetic Diseases in the Mouse." In Gene Therapy, 37–44. Berlin, Heidelberg: Springer Berlin Heidelberg, 1998. http://dx.doi.org/10.1007/978-3-642-72160-1_3.

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Gown, Allen M. "Modern Immunohistochemistry in Targeted Therapy." In Molecular Genetic Pathology, 181–96. New York, NY: Springer New York, 2012. http://dx.doi.org/10.1007/978-1-4614-4800-6_7.

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Speiser, Phyllis W., and Aubrey Milunsky. "Fetal Medical Therapy." In Genetic Disorders and the Fetus, 976–88. Hoboken, NJ, USA: John Wiley & Sons, Inc., 2015. http://dx.doi.org/10.1002/9781118981559.ch27.

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Price, Matthew J. "Genetic Considerations." In Antiplatelet Therapy in ACS and A-Fib, 100–113. Basel: S. KARGER AG, 2012. http://dx.doi.org/10.1159/000338043.

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Chaitanya, K. V. "Genetic Disorders." In Diagnostics and Gene Therapy for Human Genetic Disorders, 81–115. Boca Raton: CRC Press, 2022. http://dx.doi.org/10.1201/9781003343790-3.

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Conference papers on the topic "Genetic Therapy"

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Zou, Yi, Jiang Li, Li-Guo Zhu, Tu-Nan Chen, Kun Meng, Hua Feng, and Jian-Heng Zhao. "An initial study of genetic demyelinating mouse brain using Terahertz spectroscopy." In Cancer Imaging and Therapy. Washington, D.C.: OSA, 2016. http://dx.doi.org/10.1364/cancer.2016.jw3a.32.

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Morgan, Jeffrey R. "Genetic Strategies for Tissue Engineering." In ASME 1996 International Mechanical Engineering Congress and Exposition. American Society of Mechanical Engineers, 1996. http://dx.doi.org/10.1115/imece1996-1165.

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Abstract Recent advances in molecular genetics have resulted in the development of new technologies for the introduction and expression of genes in human somatic cells. These gene transfer technologies have given rise to a potentially new field of medical treatment known as gene therapy. Gene therapy is broadly defined as the transfer of genetic material to cells or tissues in order to achieve a therapeutic effect for inherited as well as acquired diseases. We are exploring the potential application of gene transfer technologies to the field of tissue engineering and are interested in determining if genetic modification can be used to enhance the function and/or performance of cells used as or part of biological substitutes for the restoration, maintenance or improvement of tissue function. We believe that gene transfer technologies will be an important addition to the field of tissue engineering.
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PONOMARI, Dorina. "Speech therapy assistance in the context of genetic disorders." In Ştiință și educație: noi abordări și perspective. "Ion Creanga" State Pedagogical University, 2023. http://dx.doi.org/10.46727/c.v1.24-25-03-2023.p179-184.

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Language presents a complex psychic process and many negative factors can affect it in its evolutionary path. These factors can be external psychosocial but also internal. Genetic disorders can mark to a greater or lesser extent the phenotype of the child with impact on the central nervous system and the speech apparatus. Children with some genetic conditions have difficulties in language development and often need speech therapy assistance, however early speech therapy intervention given at an early age will improve language development.
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Ashworth, Alan. "Abstract IA14: Harnessing genetic dependencies in cancer therapy." In Abstracts: AACR Precision Medicine Series: Synthetic Lethal Approaches to Cancer Vulnerabilities - May 17-20, 2013; Bellevue, WA. American Association for Cancer Research, 2013. http://dx.doi.org/10.1158/1535-7163.pms-ia14.

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Ashworth, Alan. "Abstract IA23: Harnessing genetic dependencies in cancer therapy." In Abstracts: Second AACR International Conference on Frontiers in Basic Cancer Research--Sep 14-18, 2011; San Francisco, CA. American Association for Cancer Research, 2011. http://dx.doi.org/10.1158/1538-7445.fbcr11-ia23.

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Rai, Anjani Kumar, Sumit Pundir, Anurag Shrivastava, C. Praveen Kumar, Ankit Mehta, and Pankaj Singh. "Optimizing HIV / AIDS Antiretroviral Therapy with Genetic Algorithm." In 2023 10th IEEE Uttar Pradesh Section International Conference on Electrical, Electronics and Computer Engineering (UPCON). IEEE, 2023. http://dx.doi.org/10.1109/upcon59197.2023.10434580.

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Lones, Michael A., Jane E. Alty, Phillipa Duggan-Carter, Andrew J. Turner, D. R. Stuart Jamieson, and Stephen L. Smith. "Classification and characterisation of movement patterns during levodopa therapy for parkinson's disease." In GECCO '14: Genetic and Evolutionary Computation Conference. New York, NY, USA: ACM, 2014. http://dx.doi.org/10.1145/2598394.2609852.

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Ashworth, Alan. "Abstract SY01-01: Harnessing genetic dependencies in cancer therapy." In Proceedings: AACR 102nd Annual Meeting 2011‐‐ Apr 2‐6, 2011; Orlando, FL. American Association for Cancer Research, 2011. http://dx.doi.org/10.1158/1538-7445.am2011-sy01-01.

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Higgins, Conor, Conor Ryan, Aine Kearns, and Mikael Fernstrom. "The creation and facilitation of speech and language therapy sessions for individuals with aphasia." In GECCO '14: Genetic and Evolutionary Computation Conference. New York, NY, USA: ACM, 2014. http://dx.doi.org/10.1145/2598394.2598485.

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Jonathan, Dry R., Danielle Greenawalt, Zhongwu Lai, and Carl Barrett. "Abstract B51: Tumor genetic shift post standard of care therapy." In Abstracts: AACR-NCI-EORTC International Conference: Molecular Targets and Cancer Therapeutics; November 5-9, 2015; Boston, MA. American Association for Cancer Research, 2015. http://dx.doi.org/10.1158/1535-7163.targ-15-b51.

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Reports on the topic "Genetic Therapy"

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Mohan, Subburaman. Molecular Genetic and Gene Therapy Studies of the Musculoskeletal System. Fort Belvoir, VA: Defense Technical Information Center, September 2009. http://dx.doi.org/10.21236/ada512941.

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Mohan, Subburaman. Molecular Genetic and Gene Therapy Studies of the Musculoskeletal System. Fort Belvoir, VA: Defense Technical Information Center, February 2007. http://dx.doi.org/10.21236/ada469196.

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Mohan, Subburaman. Molecular Genetic and Gene Therapy Studies of the Musculoskeletal System. Fort Belvoir, VA: Defense Technical Information Center, October 2005. http://dx.doi.org/10.21236/ada469369.

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Gilkeson, Luke. CRISPR-Cas9 Gene Therapy Review: A Novel Way to Treat Genetic Disease. Ames (Iowa): Iowa State University, May 2024. http://dx.doi.org/10.31274/cc-20240624-452.

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Robins, Diane M. Humanized Androgen Receptor Mice: A Genetic Model for Differential Response to Prostate Cancer Therapy. Fort Belvoir, VA: Defense Technical Information Center, July 2012. http://dx.doi.org/10.21236/ada582175.

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Robins, Diane. Humanized Androgen Receptor Mice: A Genetic Model for Differential Response to Prostate Cancer Therapy. Fort Belvoir, VA: Defense Technical Information Center, June 2011. http://dx.doi.org/10.21236/ada547343.

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Zhenni, Mu, Le Lei, Shen Sinan, and Tang Li. Effectiveness of integrated Chinese herbal medicine Shoutai Pill and Western medicine in the treatment of recurrent pregnancy loss: A protocol for systematic review and meta-analysis. INPLASY - International Platform of Registered Systematic Review and Meta-analysis Protocols, October 2021. http://dx.doi.org/10.37766/inplasy2021.10.0062.

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Review question / Objective: We provide a protocol to evaluate the efficacy of integrated Shoutai Pill and Western medicine to update the evaluation for the best available and security treatment for recurrent pregnancy loss(RPL). Condition being studied: Recurrent pregnancy loss (RPL) is a distinct disorder defined by two or more consecutive pregnancy failures before 20 gestational weeks infertile couples. The incidence of this disease accounts for about 1%-5% of women of reproductive age and seriously affects their physical and psychological health. At present, the known etiology of this disease mainly includes abnormal anatomic structures, genetic abnormality, endocrine disorders, prethrombotic status, abnormal immune function, infection, etc. Excluding the above factors, approximately 40-50% of RPL remain unexplained, known as unexplained recurrent pregnancy loss (URPL). At present, the main therapeutic methods of RPL are surgical therapy, preimplantation genetic diagnosis (PGD), hormone therapy, anti-infection therapy, anticoagulation, and immunoregulatory therapy, etc. However, there is no effective treatment has been identified for URPL. Therefore, we still need to investigate effective treatments to reduce pregnancy losses and maintain successful pregnancy preservation in these patients.
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Schuerger, Caroline, Steph Batalis, Katherine Quinn, Ronnie Kinoshita, Owen Daniels, and Anna Puglisi. Understanding the Global Gain-of-Function Research Landscape. Center for Security and Emerging Technology, August 2023. http://dx.doi.org/10.51593/20220035.

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Gain- and loss-of-function research have contributed to breakthroughs in vaccine development, genetic research, and gene therapy. At the same time, a subset of gain- and loss-of-function studies involve high-risk, highly virulent pathogens that could spread widely among humans if deliberately or unintentionally released. In this report, we map the gain- and loss-of-function global research landscape using a quantitative approach that combines machine learning with subject-matter expert review.
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Reding, Kerryn W. An Epidemiologic Study of Genetic Variation in Hormonal Pathways in Relation to the Effect of Hormone Replacement Therapy on Breast Cancer Risk. Fort Belvoir, VA: Defense Technical Information Center, April 2008. http://dx.doi.org/10.21236/ada486465.

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Reding, Kerryn W. An Epidemiologic Study of Genetic Variation in Hormonal Pathways in Relation to the Effect of Hormone Replacement Therapy on Breast Cancer Risk. Fort Belvoir, VA: Defense Technical Information Center, October 2008. http://dx.doi.org/10.21236/ada501717.

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