Current Trends in Clinical & Medical Sciences in Iris Publishers

Progress and Challenges in Clinical AAV Gene Therapy for Neurological and Neuromuscular Disorders Authored by Megan Baird, Maura Schwartz, Kathrin Meyer and Nicolas Wein Introduction Adeno-associated viruses (AAV) are small, replicationdefective, nonenveloped viruses from the family Parvoviridae. First discovered in the mid-1960s, AAV was thought to be a contaminant in adenovirus cultures [1,2]. Over the next 15-20 years, studies to understand basic AAV biology began, characterizing AAV elements such as genome composition and configuration [1,2]. AAV contains a linear single-stranded DNA genome containing approximately 4.7 kilobases [2,3]. As AAV is non-integrating and has a low immunogenic profile, it has become an attractive candidate for viral mediated gene therapies following the unfortunate death of Jesse Gelsinger in 1999. Jesse’s death was caused by immune complications triggered by an adenoviral based gene therapy. Despite the hesitation and fear that resulted from this even...