Adeno-associated virus vector as a platform for gene therapy delivery
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in develo** clinically …
treatment of a variety of human diseases. Recent advances in develo** clinically …
Current clinical applications of in vivo gene therapy with AAVs
JR Mendell, SA Al-Zaidy, LR Rodino-Klapac… - Molecular Therapy, 2021 - cell.com
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …
Genome-editing technologies for gene and cell therapy
ML Maeder, CA Gersbach - Molecular therapy, 2016 - cell.com
Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
Long-term effect of gene therapy on Leber's congenital amaurosis
JWB Bainbridge, MS Mehat, V Sundaram… - … England Journal of …, 2015 - Mass Medical Soc
Background Mutations in RPE65 cause Leber's congenital amaurosis, a progressive retinal
degenerative disease that severely impairs sight in children. Gene therapy can result in …
degenerative disease that severely impairs sight in children. Gene therapy can result in …
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
F Mingozzi, KA High - Blood, The Journal of the American …, 2013 - ashpublications.org
Gene therapy products for the treatment of genetic diseases are currently in clinical trials,
and one of these, an adeno-associated viral (AAV) product, has recently been licensed. AAV …
and one of these, an adeno-associated viral (AAV) product, has recently been licensed. AAV …
[HTML][HTML] Manufacturing challenges and rational formulation development for AAV viral vectors
A Srivastava, KMG Mallela, N Deorkar… - Journal of pharmaceutical …, 2021 - Elsevier
Adeno-associated virus (AAV) has emerged as a leading platform for gene delivery for
treating various diseases due to its excellent safety profile and efficient transduction to …
treating various diseases due to its excellent safety profile and efficient transduction to …
Gene therapy clinical trials worldwide to 2012–an update
SL Ginn, IE Alexander, ML Edelstein… - The journal of gene …, 2013 - Wiley Online Library
To date, over 1800 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …
been approved worldwide. Our database brings together global information on gene therapy …
Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): where are we, and how did we get here?
The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …
in Europe and the United States are landmark achievements in the history of modern …
Effect of genome size on AAV vector packaging
Z Wu, H Yang, P Colosi - Molecular Therapy, 2010 - cell.com
Adeno-associated virus (AAV) vector genomes have been limited to 5 kilobases (kb) in
length because their packaging limit was thought to be similar to the size of the parent AAV …
length because their packaging limit was thought to be similar to the size of the parent AAV …
[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges
L Kang, S **, J Wang, Z Lv, C **n, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …
central nervous system (CNS) gene therapy. AAV has already shown promising results in …