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.
A new review article in Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases. The article ...
Nov 13, 2023
0
0
Gene therapy adeno-associated viruses (AAVs)—viruses that can be engineered to deliver DNA to target cells—are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to long-term efficacy, ...
Nov 6, 2023
0
17
A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects. The study, conducted ...
Sep 28, 2023
0
11
Researchers have developed an engineered adeno-associated virus (AAV) vector that yields high transduction of brain vascular pericytes and smooth muscle cells. The study describing the characterization of this novel AAV capsid ...
Aug 24, 2023
0
7
A new study shows that total RPE65 protein levels in mice with autosomal dominant retinitis pigmentosa were doubled following subretinal delivery of adeno-associated virus (AAV)-RPE65 gene supplementation. The study is published ...
May 23, 2023
0
0
Using a process confirmation vector, researchers developed and optimized a size exclusion chromatography (SEC) with UV and multi-angle light scattering (MALS) method to measure the level of empty capsids during manufacturing. ...
May 9, 2023
0
10
A new study reports the findings of the functional evaluation of six AAV vectors in 12 preclinical models of the human liver. The study, which aimed to uncover which combination of models is the most relevant for the identification ...
May 2, 2023
0
24
A new study characterizes a bioengineered adeno-associated virus (AAV)3B capsid variant that demonstrates improved transduction to human liver cells. Another advantage of the AAV3B-V04 capsid was its significantly reduced ...
Apr 25, 2023
0
3
Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help ...
Mar 27, 2023
0
2
The use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV). The study, which showed that this strategy enabled gene ...
Mar 22, 2023
0
2
