抗肌萎缩蛋白缺陷的肌肉里重组腺相关病毒载体介导的基因转导的制约因素

Title:Restriction Factors Against Recombinant Adeno-associated Virus Vectormediated Gene Transfer in Dystrophin-deficient Muscles

Volume: 16 Issue: 3

Author(s): Jean-Baptiste Dupont

Affiliation:

关键词: 肌肉，杜氏肌肉营养不良症，腺相关病毒，基因治疗，制约因素，在体内。

摘要: Despite the unprecedented beneficial effects of rAAV gene therapy in animal models of Duchenne muscular dystrophy (DMD), the need to inject large amounts of vector in vivo to improve phenotype raises obvious biosafety concerns. While rAAV vectors generally exhibit a good safety profile, specific pathological phenotypes such as those observed in dystrophin-deficient muscles may promote immunotoxic/genotoxic effects. Increasing the therapeutic index of rAAV in DMD muscles by reducing the effective dose could be a pivotal means of ensuring efficient clinical translation. This requires a comprehensive understanding of the rAAV transduction process, which is almost always studied in non-pathological tissues or in vitro. In this review, we focus on the molecular fate of rAAV after injection, and how the individual stages of transduction could be affected in the context of DMD.

Cite this article as:

Jean-Baptiste Dupont , Restriction Factors Against Recombinant Adeno-associated Virus Vectormediated Gene Transfer in Dystrophin-deficient Muscles, Current Gene Therapy 2016; 16 (3) . https://dx.doi.org/10.2174/1566523216666160428104325