Next-generation AAV vectors—do not judge a virus (only) by its cover
C Domenger, D Grimm - Human molecular genetics, 2019 - academic.oup.com
Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …
numerous clinical trials after they have emerged as a highly promising vector for human …
In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
S Marcó, V Haurigot, F Bosch - Human Gene Therapy, 2019 - liebertpub.com
For most lysosomal storage diseases (LSDs), there is no cure. Gene therapy is an attractive
tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which …
tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which …