News-Medical.Net Gene Therapy News
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Gene therapy investigators can greatly benefit from National Gene Vector Biorepository
Mar 27, 2020 | 20:46 pmGene therapy investigators can greatly benefit from the resources and services provided by the National Gene Vector Biorepository, housed at the Indiana University School of Medicine.
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Maintaining VDR levels in pancreatic cells could protect against diabetes
Mar 27, 2020 | 12:13 pmMaintaining vitamin D receptor levels in pancreatic cells that synthesize and secrete insulin could contribute to protecting against the development of diabetes and counteract pancreatic cell damage caused by the progression of the disease.
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Study suggests potential therapeutic strategy against diabetes
Mar 26, 2020 | 20:29 pmMaintaining vitamin D receptor levels in pancreatic cells that synthesize and secrete insulin (β cells) could contribute to protecting against the development of diabetes and counteract pancreatic cell damage caused by the progression of the disease.
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High-throughput screening of AAV vector capsid libraries reveals unexpected insights
Mar 19, 2020 | 03:54 amResearchers have used high-throughput screening of adeno-associated viral (AAV) vector capsid libraries to maximize the likelihood of obtaining AAV variants with desired properties.
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Researchers identify new way to treat Danon disease using gene therapy
Mar 19, 2020 | 02:43 amDanon disease is a very rare, life-threatening condition where the fundamental biological process of removing and recycling proteins does not work.
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Recognizing CDKL5 Deficiency Disorder
Mar 17, 2020 | 10:51 amNews-Medical speaks to Dr. Dan Lavery about the LouLou Foundation, a non-profit organization dedicated to CDLK5 Deficiency Disorder.
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Fight for Sight funded researcher develops gene therapy approach for glaucoma
Mar 12, 2020 | 02:05 amA Fight for Sight funded researcher from the University of Bristol has used gene editing techniques to successfully demonstrate a gene therapy for glaucoma in the lab, in results published in Molecular Therapy in March.
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Gene therapy reverses cardiac dysfunction in mice with Barth syndrome
Mar 10, 2020 | 01:29 amBarth syndrome is a rare metabolic disease in boys caused by mutation of a gene called tafazzin or TAZ. It can cause life-threatening heart failure and also weakens the skeletal muscles, undercuts the immune response, and impairs overall growth.
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Preclinical data of Ocugen's OCU400 genetic modifier published in Nature Gene Therapy
Mar 4, 2020 | 04:32 amOcugen, Inc., a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, announced today the publication in Nature Gene Therapy of preclinical data of nuclear hormone receptor gene NR2E3 as a genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases.
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Gene therapy regenerates functional new neurons in mouse models of HD
Feb 28, 2020 | 20:54 pmHuntington's disease (HD) is a rare disease characterized by abnormal chorea movement and caused by Huntingtin (Htt) gene mutation and neurodegeneration in a brain area called striatum.
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