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 ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...
As gene therapy evolves to target additional indications—including more common indications—the choice of delivery platform is bound to become more interesting. At present, the most common delivery ...
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 ...
Adeno-associated viral vectors comprise the majority of recent gene therapy development programs due to their broad tissue-tropism and relatively low immunogenicity. Recent gene therapy approvals, ...
What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...
SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...
Viral vectors are a key component of many advanced gene therapies and vaccines, but reliable, large-scale manufacturing remains a significant challenge. Continued innovation in vector design, cell ...
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