腺相关病毒（Adeno-associated virus, AAV）是一种单链DNA病毒，目前科学界共识是它不会导致任何人类疾病。作为动物及细胞实验最重要的载体之一，重组腺相关病毒（recombinant AAV, rAAV）可携带目的基因片段，其衣壳内的基因组中编码病毒蛋白的部分完全被删除 ...

Somatic gene transfer with vectors such as adeno-associated virus (AAV) can overcome these limitations. ReGenX's AAV Vector Technology gives extremely efficient and stable gene expression in ...

Adeno-associated viruses (AAV) can be engineered so that the virus’ own genome is replaced with DNA that contains the functioning copy of the gene (known as a transgene) and a switch to turn the ...

Mydicar is under clinical development by Sardocor and currently in Phase II for Systolic Heart Failure. According to GlobalData, Phase II drugs for Systolic Heart Failure have a 67% phase transition ...

Novartis (NYSE:NVS) said a Phase 3 study of an intrathecal formulation of its gene therapy Zolgensma, also known as OAV101 IT ...

Download this compendium from The Scientist’s Creative Services Team and Polyplus, now part of Sartorius, to learn more about viral vectors for gene therapy and discover how to enhance recombinant AAV ...

ACTUS-101 is under clinical development by Asklepios BioPharmaceutical and currently in Phase II for Pompe Disease.