Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Gene therapy investigators can greatly benefit from National Gene Vector Biorepository

Gene 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.

27 Mar 2020

Maintaining VDR levels in pancreatic cells could protect against diabetes

Maintaining 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.

27 Mar 2020

Study suggests potential therapeutic strategy against diabetes

Maintaining 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.

26 Mar 2020

High-throughput screening of AAV vector capsid libraries reveals unexpected insights

Researchers have used high-throughput screening of adeno-associated viral (AAV) vector capsid libraries to maximize the likelihood of obtaining AAV variants with desired properties.

18 Mar 2020

Researchers identify new way to treat Danon disease using gene therapy

Danon disease is a very rare, life-threatening condition where the fundamental biological process of removing and recycling proteins does not work.

18 Mar 2020

Recognizing CDKL5 Deficiency Disorder

News-Medical speaks to Dr. Dan Lavery about the LouLou Foundation, a non-profit organization dedicated to CDLK5 Deficiency Disorder.

17 Mar 2020

Fight for Sight funded researcher develops gene therapy approach for glaucoma

A 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.

11 Mar 2020

Gene therapy reverses cardiac dysfunction in mice with Barth syndrome

Barth 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.

9 Mar 2020

Preclinical data of Ocugen's OCU400 genetic modifier published in Nature Gene Therapy

Ocugen, 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.

3 Mar 2020

Gene therapy regenerates functional new neurons in mouse models of HD

Huntington'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.

28 Feb 2020

New form of gene editing slows ALS progression in mice

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis - a debilitating and fatal neurological disease for which there is no cure.

26 Feb 2020

Gene-editing is more error-prone than thought, new findings suggest

The standard gene-editing tool, CRISPR-Cas9, frequently produces a type of DNA mutation that ordinary genetic analysis misses, claims new research published in the journal Proceedings of the National Academy of Sciences.

26 Feb 2020

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy, an as-yet-uncured disease that results in devastating muscle weakening and loss.

24 Feb 2020

Plant-based relatives of cholesterol could boost gene-infused nanoparticle delivery

Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol because their shape and structure help the genes get where they need to be inside cells.

20 Feb 2020

BIA Separations signs agreement with University of Zagreb to commercialize technology for viral vector purification

BIA Separations, a leading bio-chromatography development and manufacturing company, announced it has signed a licensing agreement with the University of Zagreb to commercialize a novel elution method that helps preserve the integrity, infectivity and potency of viruses during immunoaffinity purification.

19 Feb 2020

Research suggests new approach to block tau pathology in FTLD, Alzheimer's

The protein β-arrestin-2 increases the accumulation of neurotoxic tau tangles, a cause several forms of dementia, by interfering with removal of excess tau from the brain, a new study by the University of South Florida Health Morsani College of Medicine found.

17 Feb 2020

Gene Therapy News and Research

Further Reading

Gene therapy investigators can greatly benefit from National Gene Vector Biorepository

Maintaining VDR levels in pancreatic cells could protect against diabetes

Study suggests potential therapeutic strategy against diabetes

High-throughput screening of AAV vector capsid libraries reveals unexpected insights

Researchers identify new way to treat Danon disease using gene therapy

Recognizing CDKL5 Deficiency Disorder

Fight for Sight funded researcher develops gene therapy approach for glaucoma

Gene therapy reverses cardiac dysfunction in mice with Barth syndrome

Preclinical data of Ocugen's OCU400 genetic modifier published in Nature Gene Therapy

Gene therapy regenerates functional new neurons in mouse models of HD

New form of gene editing slows ALS progression in mice

Gene-editing is more error-prone than thought, new findings suggest

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Plant-based relatives of cholesterol could boost gene-infused nanoparticle delivery

BIA Separations signs agreement with University of Zagreb to commercialize technology for viral vector purification

Research suggests new approach to block tau pathology in FTLD, Alzheimer's

Neuronal necrosis occurs much earlier in Alzheimer’s disease progression than previously thought

InquireFirst announces Cross-Border Science Journalism Project for Latin America

Researchers investigate methods to 3D bioprint tissues for craniomaxillofacial reconstruction

Outcomes-based drug reimbursement may be on the horizon, report says

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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