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.
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
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17
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. ...
Sep 10, 2021
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72
Researchers at KU Leuven in Belgium have developed a technique to make sheep produce new antibodies simply by injecting the DNA building blocks. This approach is much cheaper and more efficient than producing antibodies industrially ...
Sep 11, 2019
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69
Adoptive cell-therapies, in which a patient's own immune cells are used to recognize and target tumors, have shown breakthrough results for patients with certain B-cell malignancies, or cancers of the blood, but this therapy ...
Sep 14, 2016
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115
An Australian scientist said Wednesday he had discovered a way to turn the HIV virus against itself in human cells in the laboratory, in an important advance in the quest for an AIDS cure.
Jan 16, 2013
22
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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
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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
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7
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
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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
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3
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
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2
