[PDF][PDF] CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
[HTML][HTML] Adeno-associated viral vectors in neuroscience research
DL Haggerty, GG Grecco, KC Reeves… - … Therapy-Methods & …, 2020 - cell.com
Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in
neuroscience research studies for interrogating cellular and neurocircuit functions and …
neuroscience research studies for interrogating cellular and neurocircuit functions and …
Can adeno-associated viral vectors deliver effectively large genes?
P Tornabene, I Trapani - Human gene therapy, 2020 - liebertpub.com
Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …
Dual and triple AAV delivery of large therapeutic gene sequences into the inner ear
O Akil - Hearing Research, 2020 - Elsevier
Abstract Adeno-associated virus (AAV)-mediated gene therapy has evolved from the bench
to the bedside, and is now considered the therapy of choice for certain inherited diseases …
to the bedside, and is now considered the therapy of choice for certain inherited diseases …
[HTML][HTML] Promise and progress of an HIV-1 cure by adeno-associated virus vector delivery of anti-HIV-1 biologics
MR Gardner - Frontiers in Cellular and Infection Microbiology, 2020 - frontiersin.org
Despite the success of antiretroviral therapy (ART) at suppressing HIV-1 infection, a cure
that eradicates all HIV-1-infected cells has been elusive. The latent viral reservoir remains …
that eradicates all HIV-1-infected cells has been elusive. The latent viral reservoir remains …
Biodistribution analysis of striatal and cerebellar administration of modified adeno-associated viral vectors in normal mice
IT Afonso - 2020 - sapientia.ualg.pt
Os vírus adeno-associados (AAV) têm 25 nm de diâmetro e transportam uma molécula de
DNA de cadeia simples. O seu genoma está flanqueado por duas repetições terminais …
DNA de cadeia simples. O seu genoma está flanqueado por duas repetições terminais …
Genome Engineering Goes Viral: Repurposing of Adeno-associated Viral Vectors for CRISPR-mediated in Vivo Genome Engineering
RR Ibraheim - 2020 - repository.escholarship.umassmed …
One of the major challenges facing medicine and drug discovery is the large number of
genetic diseases caused by inherited mutations leading to a toxic gain-of-function, or loss-of …
genetic diseases caused by inherited mutations leading to a toxic gain-of-function, or loss-of …
Characterization of AAVrh. 10 as a vector to deliver long-lasting genetic constructs into neurons of neonatal mice following intravenous injection
M Martin - 2020 - mspace.lib.umanitoba.ca
Prion diseases are a fatal neurodegenerative disease caused by the misfolding of the
cellular prion protein, PrPC into an infectious isoform, PrPSc. Accumulation of this infectious …
cellular prion protein, PrPC into an infectious isoform, PrPSc. Accumulation of this infectious …
Elucidating the Factors Determining Efficient Transduction of Human Airway Epithelia by AAV Gene Therapy Vectors
BA Hamilton - 2020 - search.proquest.com
Vectors derived from adeno-associated virus (AAV) are FDA approved to transfer genes to
the retina for treatment of Leber's congenital amaurosis. AAVs have also been proposed to …
the retina for treatment of Leber's congenital amaurosis. AAVs have also been proposed to …