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New Rochelle, NY, January 19, 2021 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 concerns, and strategies to overcome these challenges are highlighted in the peer-reviewed journal
Human Gene Therapy. Click here to read the full-text article free on the
Human Gene Therapy website. Currently, the field is undergoing rapid development with a number of competing gene editing strategies, including allele-specific knock-down, base editing, prime editing, and RNA editing, are under investigation. Each offers a different balance of on-target editing efficiency versus off-target risks, state Kanmin Xue, Unive
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IMAGE: Provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. view more
Credit: Mary Ann Liebert, Inc., publishers
New Rochelle, NY, January 15, 2021 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. Study design and results are presented in the peer-reviewed journal
Human Gene Therapy. Click here to read the full-text article free on the
Human Gene Therapy website through February 15, 2021.
AAV capsids were labeled with I-124 and delivered using two routes of administration: intravenous and directly into the cerebrospinal fluid (CSF). Biodistribution was measured by quantitative positron emission tomography (PET) at 1, 24, 48, and 72 hours after AAV administration. Two AAV vectors - AAVrsh.10 and AAV9 - we
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IMAGE: journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. view more
Credit: Mary Ann Liebert, Inc., publishers
New Rochelle, NY, December 15, 2020 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. Click here to read the full-text article free on the
Human Gene Therapy website through January 15, 2021.
Reduced uterine blood flow and lack of bioavailable VEGF due to placental insufficiency is a major cause of severe fetal growth restriction (FGR). This is untreatable and causes serious neonatal morbidity and death.
Date Time
Gene Therapy for Placental Insufficiency Moves Toward Clinic
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. Click here to read the full-text article free on the Human Gene Therapy website through January 15, 2021.
Reduced uterine blood flow and lack of bioavailable VEGF due to placental insufficiency is a major cause of severe fetal growth restriction (FGR). This is untreatable and causes serious neonatal morbidity and death.
Anna David, UCL Institute for Women’s Health, and colleagues tested different VEGF isoforms on endothelial cells from four species, including from human umbilical vein. The results support the use of the best performing VEGF isoform “in a human clinical trial for FGR caused by placental insufficiency.”