Viral vector platforms within the gene therapy landscape
Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …
transitions. It has seen great strides in combating human disease, has given hope to patients …
AAV vector immunogenicity in humans: a long journey to successful gene transfer
HC Verdera, K Kuranda, F Mingozzi - Molecular Therapy, 2020 - cell.com
Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …
Overcoming innate immune barriers that impede AAV gene therapy vectors
M Muhuri, Y Maeda, H Ma, S Ram… - The Journal of …, 2021 - Am Soc Clin Investig
The field of gene therapy has made considerable progress over the past several years.
Adeno-associated virus (AAV) vectors have emerged as promising and attractive tools for in …
Adeno-associated virus (AAV) vectors have emerged as promising and attractive tools for in …
Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases
Recently, adeno-associated virus (AAV)-mediated gene therapies have attracted clinical
interest for treating neurodegenerative diseases including spinal muscular atrophy (SMA) …
interest for treating neurodegenerative diseases including spinal muscular atrophy (SMA) …
Understanding and tackling immune responses to adeno-associated viral vectors
H Costa-Verdera, C Unzu, E Valeri, S Adriouch… - Human Gene …, 2023 - liebertpub.com
As the clinical experience in adeno-associated viral (AAV) vector-based gene therapies is
expanding, the necessity to better understand and control the host immune responses is …
expanding, the necessity to better understand and control the host immune responses is …
Pig Models in Retinal Research and Retinal Disease
MA McCall - Cold Spring Harbor Perspectives in …, 2024 - perspectivesinmedicine.cshlp.org
The pig has been used as a large animal model in biomedical research for many years and
its use continues to increase because induced mutations phenocopy several inherited …
its use continues to increase because induced mutations phenocopy several inherited …
Enhancing Safety and Efficacy of Genome Editing In Vivo with Compact Cas9 and Guide Chemical Modification
H Zhang - 2024 - repository.escholarship.umassmed …
Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins
(CRISPR/Cas) have revolutionized science and medicine. However, applying the …
(CRISPR/Cas) have revolutionized science and medicine. However, applying the …