Pancreatic cancer is an incurable form of cancer, and gene therapies are currently in clinical testing to treat this deadly disease. A comprehensive review of the gene and cell biotherapies in development to combat pancreatic ...
Mar 6, 2023
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Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in Human Gene Therapy.
Feb 22, 2023
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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 ...
Feb 1, 2022
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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 ...
Jan 25, 2022
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Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A. This pharmacology study, performed in a mouse model and designed to support the initiation of ...
Dec 16, 2021
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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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Norwegian biologist Gro Amdam writes this in an email to ScienceNorway from the U.S., where she is professor at Arizona State University.
Apr 23, 2021
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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 ...
Jan 20, 2021
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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. ...
Jan 15, 2021
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A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy.
Dec 16, 2020
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