Semantic Scholar Open Access 2019 287 sitasi

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.

E. Hudry L. Vandenberghe

Lihat Sumber DOI

Abstrak

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly considered for rare inherited disorders, gene therapy may open treatment opportunities for more challenging and complex diseases such as Alzheimer's or Parkinson's disease. Today, examples of striking clinical proof of concept, the first gene therapy drugs coming onto the market, and the emergence of powerful new molecular tools have broadened the number of avenues to target neurological disorders but have also highlighted safety concerns and technology gaps. The vector of choice for many nervous system targets currently is the adeno-associated viral (AAV) vector due to its desirable safety profile and strong neuronal tropism. In aggregate, the clinical success, the preclinical potential, and the technological innovation have made therapeutic AAV drug development a reality, particularly for nervous system disorders. Here, we discuss the rationale, opportunities, limitations, and progress in clinical AAV gene therapy.

Topik & Kata Kunci

Medicine

Penulis (2)

E. Hudry

L. Vandenberghe

Format Sitasi

APA MLA BibTeX

Hudry, E., Vandenberghe, L. (2019). Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.. https://doi.org/10.1016/j.neuron.2019.02.017

Akses Cepat

Lihat di Sumber doi.org/10.1016/j.neuron.2019.02.017

Informasi Jurnal

Tahun Terbit: 2019
Bahasa: en
Total Sitasi: 287×
Sumber Database: Semantic Scholar
DOI: 10.1016/j.neuron.2019.02.017
Akses: Open Access ✓