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/
Impact factor
4.218 ()

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Genetics

Treating cystic fibrosis with mRNA therapy or CRISPR

The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible ...

Genetics

Engineered capsids for efficient gene delivery to the eye

A rational design approach created novel variants of adeno-associated viral (AAV) capsids. These have improved transduction properties in the mouse retina and cornea. as reported in the peer-reviewed journal Human Gene Therapy.

Genetics

Gene therapy targets inner retina to combat blindness

Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease. Adeno-associated viral (AAV)-mediated expression of the human CLN3 gene led to significant survival ...

Genetics

Concern following gene therapy adverse events

The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...

Genetics

Using gene therapy to treat chronic traumatic encephalopathy

A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study, which demonstrated the effectiveness of direct delivery of gene ...

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