[PDF][PDF] Therapeutic AAV gene transfer to the nervous system: a clinical reality
E Hudry, LH Vandenberghe - Neuron, 2019 - cell.com
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 …
considered for rare inherited disorders, gene therapy may open treatment opportunities for …
[HTML][HTML] Gene therapy for Duchenne muscular dystrophy
N Elangkovan, G Dickson - Journal of neuromuscular …, 2021 - content.iospress.com
Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …
Delivery and specificity of CRISPR/Cas9 genome editing technologies for human gene therapy
Genome editing using the clustered regularly interspaced short palindromic repeats
(CRISPR)/CRISPR associated 9 (Cas9) technology is revolutionizing the study of gene …
(CRISPR)/CRISPR associated 9 (Cas9) technology is revolutionizing the study of gene …
[HTML][HTML] A comprehensive review of retinal gene therapy
Blindness, although not life threatening, is a debilitating disorder for which few, if any
treatments exist. Ocular gene therapies have the potential to profoundly improve the quality …
treatments exist. Ocular gene therapies have the potential to profoundly improve the quality …
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
J McIntosh, PJ Lenting, C Rosales… - Blood, The Journal …, 2013 - ashpublications.org
Recombinant adeno-associated virus (rAAV) vectors encoding human factor VIII (hFVIII)
were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV …
were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV …
Effective delivery of large genes to the retina by dual AAV vectors
Retinal gene therapy with adeno‐associated viral (AAV) vectors is safe and effective in
humans. However, AAV's limited cargo capacity prevents its application to therapies of …
humans. However, AAV's limited cargo capacity prevents its application to therapies of …
Viral vectors for gene delivery to the central nervous system
TB Lentz, SJ Gray, RJ Samulski - Neurobiology of disease, 2012 - Elsevier
The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …
Expressing transgenes that exceed the packaging capacity of adeno-associated virus capsids
K Chamberlain, JM Riyad, T Weber - Human gene therapy methods, 2016 - liebertpub.com
Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …
[HTML][HTML] Gene therapy and genome surgery in the retina
JE DiCarlo, VB Mahajan… - The Journal of clinical …, 2018 - Am Soc Clin Investig
Precision medicine seeks to treat disease with molecular specificity. Advances in genome
sequence analysis, gene delivery, and genome surgery have allowed clinician-scientists to …
sequence analysis, gene delivery, and genome surgery have allowed clinician-scientists to …
[HTML][HTML] Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry
LF Barnes, BE Draper, YT Chen, TW Powers… - … Therapy-Methods & …, 2021 - cell.com
Recombinant adeno-associated virus (rAAV) has emerged as an important gene therapy
vector with many clinical trials currently in progress. Analytical characterization and …
vector with many clinical trials currently in progress. Analytical characterization and …