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 …
[HTML][HTML] Current strategies for brain drug delivery
X Dong - Theranostics, 2018 - ncbi.nlm.nih.gov
The blood-brain barrier (BBB) has been a great hurdle for brain drug delivery. The BBB in
healthy brain is a diffusion barrier essential for protecting normal brain function by impeding …
healthy brain is a diffusion barrier essential for protecting normal brain function by impeding …
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 …
Viral vectors for therapy of neurologic diseases
SR Choudhury, E Hudry, CA Maguire… - …, 2017 - Elsevier
Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …
contributor to global disease burden with a shockingly large associated economic cost …
Global CNS transduction of adult mice by intravenously delivered rAAVrh. 8 and rAAVrh. 10 and nonhuman primates by rAAVrh. 10
Some recombinant adeno-associated viruses (rAAVs) can cross the neonatal blood–brain
barrier (BBB) and efficiently transduce cells of the central nervous system (CNS). However …
barrier (BBB) and efficiently transduce cells of the central nervous system (CNS). However …
Biology of adeno-associated viral vectors in the central nervous system
Gene therapy is a promising approach for treating a spectrum of neurological and
neurodegenerative disorders by delivering corrective genes to the central nervous system …
neurodegenerative disorders by delivering corrective genes to the central nervous system …
BBB-crossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment
The presence of the blood-brain barrier (BBB) remains a challenge in the treatment of
central nervous system (CNS) diseases, as it hinders the infiltration of many therapeutic …
central nervous system (CNS) diseases, as it hinders the infiltration of many therapeutic …
Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector
Effective gene delivery to the central nervous system (CNS) is vital for development of novel
gene therapies for neurological diseases. Adeno-associated virus (AAV) vectors have …
gene therapies for neurological diseases. Adeno-associated virus (AAV) vectors have …
In vivo selection yields AAV-B1 capsid for central nervous system and muscle gene therapy
SR Choudhury, Z Fitzpatrick, AF Harris, SA Maitland… - Molecular Therapy, 2016 - cell.com
Adeno-associated viral (AAV) vectors have shown promise as a platform for gene therapy of
neurological disorders. Achieving global gene delivery to the central nervous system (CNS) …
neurological disorders. Achieving global gene delivery to the central nervous system (CNS) …
Empty virions in AAV8 vector preparations reduce transduction efficiency and may cause total viral particle dose-limiting side effects
Empty virions are inadvertent by-products of recombinant adeno-associated virus (rAAV)
packaging process, resulting in vector lots with mixtures of full and empty virions at variable …
packaging process, resulting in vector lots with mixtures of full and empty virions at variable …