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Journal articles on the topic 'Genetic Therapy'

Author: Grafiati
Published: 4 June 2021
Last updated: 29 July 2024

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1

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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2

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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3

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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4

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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5

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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6

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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7

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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8

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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9

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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10

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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11

Lamba, Jatinder K. "Genetic factors influencing cytarabine therapy." Pharmacogenomics 10, no. 10 (October 2009): 1657–74. http://dx.doi.org/10.2217/pgs.09.118.

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12

Wilson, S., and D. Yeomans. "Genetic therapy for pain management." Neurology Bulletin XXXIII, no. 1-2 (May 15, 2001): 112. http://dx.doi.org/10.17816/nb79782.

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13

Kohn, Donald B., W. French Anderson, and R. Michael Blaese. "Gene Therapy for Genetic Diseases." Cancer Investigation 7, no. 2 (January 1989): 179–92. http://dx.doi.org/10.3109/07357908909038283.

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14

Younes, Mamoun, Juan Lechago, and Atilla Ertan. "Photodynamic therapy and genetic abnormalities." Gastroenterology 120, no. 4 (March 2001): 1065. http://dx.doi.org/10.1053/gast.2001.23102.

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15

Blau, C. Anthony, and George Stamatoyannopoulos. "Preemptive therapy for genetic disease." Nature Medicine 2, no. 2 (February 1996): 161–62. http://dx.doi.org/10.1038/nm0296-161.

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16

Caskey, C. Thomas. "Genetic Therapy: Somatic Gene Transplants." Hospital Practice 22, no. 8 (August 15, 1987): 181–98. http://dx.doi.org/10.1080/21548331.1987.11703293.

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17

Loughlin, Kevin R. "Renal cancer: Genetic directed therapy." Urologic Oncology: Seminars and Original Investigations 27, no. 2 (March 2009): 130. http://dx.doi.org/10.1016/j.urolonc.2008.08.004.

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18

Wilson, Steven P., and David C. Yeomans. "Genetic therapy for pain management." Current Review of Pain 4, no. 6 (December 2000): 445–50. http://dx.doi.org/10.1007/s11916-000-0068-5.

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19

Penticuff, Joy. "Ethical Issues in Genetic Therapy." Journal of Obstetric, Gynecologic & Neonatal Nursing 23, no. 6 (July 1994): 498–501. http://dx.doi.org/10.1111/j.1552-6909.1994.tb01911.x.

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20

Desnick, Robert J., and Edward H. Schuchman. "Gene therapy for genetic diseases." Pediatrics International 40, no. 3 (June 1998): 191–203. http://dx.doi.org/10.1111/j.1442-200x.1998.tb01912.x.

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21

Poluri, Ananthalakshmi, Marc van Maanen, and Richard E. Sutton. "Genetic therapy for HIV/AIDS." Expert Opinion on Biological Therapy 3, no. 6 (September 2003): 951–63. http://dx.doi.org/10.1517/14712598.3.6.951.

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22

Idle, Jeffrey R. "Genetic factors in drug therapy." Trends in Pharmacological Sciences 15, no. 8 (August 1994): 309. http://dx.doi.org/10.1016/0165-6147(94)90015-9.

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23

Sullenger, Bruce A. "Targeted genetic repair: an emerging approach to genetic therapy." Journal of Clinical Investigation 112, no. 3 (August 1, 2003): 310–11. http://dx.doi.org/10.1172/jci200319419.

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24

RESNIK, DAVID B. "The Moral Significance of the Therapy-Enhancement Distinction in Human Genetics." Cambridge Quarterly of Healthcare Ethics 9, no. 3 (July 2000): 365–77. http://dx.doi.org/10.1017/s0963180100903086.

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The therapy-enhancement distinction occupies a central place in contemporary discussions of human genetics and has been the subject of much debate. At a recent conference on gene therapy policy, scientists predicted that within a few years researchers will develop techniques that can be used to enhance human traits. In thinking about the morality of genetic interventions, many writers have defended somatic gene therapy, and some have defended germline gene therapy, but only a handful of writers defend genetic enhancement, or even give it a fair hearing. The mere mention of genetic enhancement makes many people cringe and brings to mind the Nazi eugenics programs, Aldous Huxley's Brave New World, “The X-Files,” or the recent movie “Gattaca.” Although many people believe that gene therapy has morally legitimate medical uses, others regard genetic enhancement as morally problematic or decidedly evil.
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25

Wei, Chunjiang, Weijia Kong, and Zuhong He. "Application of gene therapy in auditory system diseases." STEMedicine 1, no. 1 (January 2, 2020): e17. http://dx.doi.org/10.37175/stemedicine.v1i1.17.

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How to prevent and treat auditory related diseases through genetic intervention is a hotspot in the field of hearing research in recent years. With the development of molecular biology, molecular genetics, genetic engineering, etc., especially, gene regulation has made a major breakthrough in the research of inner ear hair cell regeneration in recent years, which may provide us with a novel and efficient way to treat auditory related diseases. This review includes the latest research on gene therapy in hereditary deafness, drug deafness, aging-related hearing loss, and noise-related hearing loss.
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26

Maltsev, S. V., A. I. Safina, and T. V. Mihajlova. "Гипофосфатемический рахит у детей — клинические и генетические аспекты, подходы к терапии." Practical medicine 19, no. 1 (2021): 38–49. http://dx.doi.org/10.32000/2072-1757-2021-1-38-49.

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Hypophosphatemic rickets (phosphate-diabetes) is a group of diseases associated with a defect in the reabsorption of phosphates in the proximal tubules, manifested by phosphaturia, hypophosphatemia and rickets deformities of the skeleton bones. Phosphate-diabetes has different genetic variants that determine the nature and severity of clinical manifestations. X-linked dominant hypophosphatemic rickets occurs most often (in 50-90% of cases). For the diagnosis, along with clinical characteristics, an important role is assigned to the study of partial renal functions, with the determination of clearance, excreted fraction of calcium and phosphates, as well as other indicators of calcium-phosphorus metabolism. Molecular genetic research helps to determine the form of the disease. Therapy for hypophosphatemic rickets should be differentiated depending on the type of disease. The timely appointment of an adequate pathogenetic therapy helps to slow down the formation of rickety deformities of the skeleton, positive growth dynamics, and an increase in physical activity.
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27

Zimina, Zimina Yu D., Gerasimenko O. N. Gerasimenko, Voronina E. N. Voronina, and Tolmacheva A. A. Tolmacheva. "Clinical-functional and molecular-genetic peculiarities of patients with chronic heart failure." Therapy 4_2024 (June 24, 2024): 41–49. http://dx.doi.org/10.18565/therapy.2024.4.41-49.

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28

Rassekh, S. Rod, Michael Rieder, and Geert ‘t Jong. "Gene-based drug therapy in children." Paediatrics & Child Health 28, no. 4 (June 6, 2023): 241–45. http://dx.doi.org/10.1093/pch/pxad002.

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Abstract The past two decades have seen enormous advancements in medical knowledge around the role of genetic factors of variability, both in human disease and drug response. This knowledge is increasingly being translated into guidelines that inform drug dosing, monitoring for efficacy and safety, and determining the suitability of specific agents to treat patients. Health Canada and the U.S. Food and Drug Administration have recommended using genetic information to guide dosing for more than 20 drugs. There are no current, comprehensive paediatric guidelines to assist health care professionals in the use of genetics to inform medication dosing, safety, and efficacy in children, and such guidance is urgently needed. This statement helps to guide clinician understanding of the role of pharmacogenetics and how to use this information when prescribing medications in paediatrics.
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29

Roh, Eun, Anjani Darai, Jae Kyung, Hyemin Choi, Su Kwon, Basanta Bhujel, Kyoung Kim, and Inbo Han. "Genetic Therapy for Intervertebral Disc Degeneration." International Journal of Molecular Sciences 22, no. 4 (February 4, 2021): 1579. http://dx.doi.org/10.3390/ijms22041579.

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Intervertebral disc (IVD) degeneration can cause chronic lower back pain (LBP), leading to disability. Despite significant advances in the treatment of discogenic LBP, the limitations of current treatments have sparked interest in biological approaches, including growth factor and stem cell injection, as new treatment options for patients with chronic LBP due to IVD degeneration (IVDD). Gene therapy represents exciting new possibilities for IVDD treatment, but treatment is still in its infancy. Literature searches were conducted using PubMed and Google Scholar to provide an overview of the principles and current state of gene therapy for IVDD. Gene transfer to degenerated disc cells in vitro and in animal models is reviewed. In addition, this review describes the use of gene silencing by RNA interference (RNAi) and gene editing by the clustered regularly interspaced short palindromic repeats (CRISPR) system, as well as the mammalian target of rapamycin (mTOR) signaling in vitro and in animal models. Significant technological advances in recent years have opened the door to a new generation of intradiscal gene therapy for the treatment of chronic discogenic LBP.
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30

Feske, Steven K. "Genetic Profile–Guided Therapy for Stroke?" New England Journal of Medicine 385, no. 27 (December 30, 2021): 2576–77. http://dx.doi.org/10.1056/nejme2116888.

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31

Carpena, Nathanial T., and Min Young Lee. "Genetic Hearing Loss and Gene Therapy." Genomics & Informatics 16, no. 4 (December 31, 2018): e20. http://dx.doi.org/10.5808/gi.2018.16.4.e20.

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32

Barlow, David H., and Stephen Kennedy. "Endometriosis: New Genetic Approaches and Therapy." Annual Review of Medicine 56, no. 1 (February 2005): 345–56. http://dx.doi.org/10.1146/annurev.med.55.091902.103805.

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33

Wood, John N. "II. Genetic approaches to pain therapy." American Journal of Physiology-Gastrointestinal and Liver Physiology 278, no. 4 (April 1, 2000): G507—G512. http://dx.doi.org/10.1152/ajpgi.2000.278.4.g507.

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New analgesic drugs are necessary because a number of pain states are untreatable. Genetic approaches to the identification of analgesic drug targets include mapping genes involved in human pain perception (e.g., trkA involved in hereditary neuropathies), identifying regulators of sensory neuron function in simple multicellular organisms and then investigating the activity of their mammalian homologs (e.g., POU domain transcription factors that specify sensory cell fate), as well as difference, expression, and homology cloning of receptors, ion channels, and transcription factors present in sensory neurons. After target validation through the construction of null mutant mice, high-throughput cell-based screens can be used to identify potential drug candidates. As a result of these approaches, a number of receptors and ion channels present in sensory neurons such as voltage-gated sodium channels [sensory neuron specific (SNS) and Na channel novel] and ATP-gated (P2X3), capsaicin-gated [vanilloid receptor 1(VR1)], and proton-gated [acid-sensing ion channel (ASIC)] channels are now under investigation as potential new analgesic drug targets.
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34

Bowers, W. J., X. O. Breakefield, and M. Sena-Esteves. "Genetic therapy for the nervous system." Human Molecular Genetics 20, R1 (March 23, 2011): R28—R41. http://dx.doi.org/10.1093/hmg/ddr110.

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35

Kinnon, C., and R. J. Levinsky. "Somatic gene therapy for genetic disease." Archives of Disease in Childhood 65, no. 1 (January 1, 1990): 72–73. http://dx.doi.org/10.1136/adc.65.1.72.

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36

DEISSEROTH, ALBERT B., ELIE G. HANANIA, SIQING FU, DAVID CLAXTON, MICHAEL ANDREEFF, RICHARD CHAMPLIN, JOHN KAVANAGH, et al. "Genetic Therapy of Human Neoplastic Disease." Journal of Hematotherapy 2, no. 3 (January 1993): 373–75. http://dx.doi.org/10.1089/scd.1.1993.2.373.

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37

Lawler, S. E., P. P. Peruzzi, and E. A. Chiocca. "Genetic strategies for brain tumor therapy." Cancer Gene Therapy 13, no. 3 (September 2, 2005): 225–33. http://dx.doi.org/10.1038/sj.cgt.7700886.

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38

JOHNSON, KATE. "Genetic Diabetes Responds to Tailored Therapy." Pediatric News 39, no. 4 (April 2005): 36–37. http://dx.doi.org/10.1016/s0031-398x(05)70123-5.

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39

Hynicka, Lauren M., William D. Cahoon, and Bonny L. Bukaveckas. "Genetic Testing for Warfarin Therapy Initiation." Annals of Pharmacotherapy 42, no. 9 (July 29, 2008): 1298–303. http://dx.doi.org/10.1345/aph.1l127.

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40

Bennett, C. Frank, and Stanislaw M. Stepkowski. "Genetic therapy for transplant vascular sclerosis." Transplant Immunology 5, no. 4 (December 1997): 299–302. http://dx.doi.org/10.1016/s0966-3274(97)80012-3.

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41

Wheldon, TE, RJ Mairs, and M. Boyd. "Genetic enhancement of radionuclide cancer therapy." Lancet 354, no. 9194 (December 1999): 1999–2000. http://dx.doi.org/10.1016/s0140-6736(05)76773-8.

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42

Barisani, Donatella, Richard M. Green, and John L. Gollan. "Genetic Hemochromatosis: Pathogenesis, Diagnosis, and Therapy." Digestive Diseases 14, no. 5 (1996): 304–15. http://dx.doi.org/10.1159/000171561.

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43

Gorovitz, Samuel. "Genetic therapy: Mapping the ethical challanges." Philosophia 25, no. 1-4 (April 1997): 83–97. http://dx.doi.org/10.1007/bf02380027.

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44

Guskov, E. P., T. P. Shkurat, E. I. Shimanskaja, and S. I. Guskova. "Genetic effects of hyperbaric oxygen therapy." Mutation Research/Genetic Toxicology 241, no. 4 (August 1990): 341–47. http://dx.doi.org/10.1016/0165-1218(90)90063-8.

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45

Bahcall, Orli G. "Genetic risk and statin preventative therapy." Nature Reviews Genetics 16, no. 4 (March 18, 2015): 194. http://dx.doi.org/10.1038/nrg3928.

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46

Young, Annie, and David J. Kerr. "Genetic and immunological therapy for cancer." Journal of the Royal Society of Medicine 93, no. 1 (January 2000): 10–14. http://dx.doi.org/10.1177/014107680009300104.

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47

Khavari, Paul A. "Gene Therapy for Genetic Skin Disease." Journal of Investigative Dermatology 110, no. 4 (April 1998): 462–67. http://dx.doi.org/10.1038/jid.1998.3.

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48

Tanner, Elizabeth J., Karla A. Kirkegaard, and Leor S. Weinberger. "Exploiting Genetic Interference for Antiviral Therapy." PLOS Genetics 12, no. 5 (May 5, 2016): e1005986. http://dx.doi.org/10.1371/journal.pgen.1005986.

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49

MacKenzie, Alex. "Genetic therapy for spinal muscular atrophy." Nature Biotechnology 28, no. 3 (March 2010): 235–37. http://dx.doi.org/10.1038/nbt0310-235.

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50

Khavari, Paul A. "Gene Therapy for Genetic Skin Disease." Journal of Investigative Dermatology 110, no. 4 (April 1998): 462. http://dx.doi.org/10.1046/j.1523-1747.1998.00157.x.

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