Human Gene Therapy

Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.

Website
http://www.liebertpub.com/overview/human-gene-therapybr-and-human-gene-therapy-methods/19/
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Medications

Dexamethasone enhances transgene expression

Dexamethasone, a glucocorticoid with anti-inflammatory and immunosuppressive effects, can transiently increase the expression of therapeutic genes delivered using adeno-associated virus (AAV) vectors. A new study, which showed ...

Genetics

Gene therapy for the treatment of Huntington's disease

Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington's disease after a single administration. An adeno-associated virus (AAV) gene ...

Oncology & Cancer

A T-cell power-up for tumor treatments

Cancer is the second leading cause of death in the United States. But in recent years, a revolutionary therapy has brought new hope in the fight against the disease that takes the lives of nearly 600,000 Americans each year. ...

Genetics

Genome editing to treat human retinal degeneration

Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Technological advances in gene editing, continuing safety ...

Genetics

Biodistribution of AAV gene transfer vectors in nonhuman primate

The biodistribution of adeno-associated virus (AAV) gene transfer vectors can be measured in nonhuman primates using a new method. The method quantifies whole-body and organ-specific AAV capsids from 1 to 72 hours after administration. ...

Genetics

AAV capsid-promoter interactions in the non-human primate brain

The phenomenon of AAV capsid-promoter interaction recently seen in the rat central nervous system has now been shown to occur in the non-human primate brain. This interaction can directly determine cell-specific transgene ...

Genetics

Gene therapy startup to increase AAV gene therapy efficiency

Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project ...

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