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 …
Production, processing, and characterization of synthetic AAV gene therapy vectors
J El Andari, D Grimm - Biotechnology journal, 2021 - Wiley Online Library
Over the last two decades, gene therapy vectors based on wild‐type Adeno‐associated
viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into …
viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into …
Human and insect cell-produced recombinant adeno-associated viruses show differences in genome heterogeneity
NT Tran, E Lecomte, S Saleun, S Namkung… - Human Gene …, 2022 - liebertpub.com
In the past two decades, adeno-associated virus (AAV) vector manufacturing has made
remarkable advancements to meet large-scale production demands for preclinical and …
remarkable advancements to meet large-scale production demands for preclinical and …
Methods matter: standard production platforms for recombinant AAV produce chemically and functionally distinct vectors
NG Rumachik, SA Malaker, N Poweleit… - … therapy Methods & …, 2020 - cell.com
Different approaches are used in the production of recombinant adeno-associated virus
(rAAV). The two leading approaches are transiently transfected human HEK293 cells and …
(rAAV). The two leading approaches are transiently transfected human HEK293 cells and …
AAV-genome population sequencing of vectors packaging CRISPR components reveals design-influenced heterogeneity
NT Tran, C Heiner, K Weber, M Weiand… - … Therapy Methods & …, 2020 - cell.com
The gene therapy field has been galvanized by two technologies that have revolutionized
treating genetic diseases: vectors based on adeno-associated viruses (AAVs), and clustered …
treating genetic diseases: vectors based on adeno-associated viruses (AAVs), and clustered …
Stowaways in the cargo: Contaminating nucleic acids in rAAV preparations for gene therapy.
MA Brimble, SM Winston, AM Davidoff - Molecular Therapy, 2023 - cell.com
Recombinant AAV (rAAV) is the most used delivery vector for clinical gene therapy.
However, many issues must be addressed before safer and more widespread …
However, many issues must be addressed before safer and more widespread …
Cryo-EM-based discovery of a pathogenic parvovirus causing epidemic mortality by black wasting disease in farmed beetles
We use cryoelectron microscopy (cryo-EM) as a sequence-and culture-independent
diagnostic tool to identify the etiological agent of an agricultural pandemic. For the past 4 …
diagnostic tool to identify the etiological agent of an agricultural pandemic. For the past 4 …
Nanopore sequencing of native adeno-associated virus (AAV) single-stranded DNA using a transposase-based rapid protocol
MT Radukic, D Brandt, M Haak… - NAR Genomics and …, 2020 - academic.oup.com
Next-generation sequencing of single-stranded DNA (ssDNA) enables transgene
characterization of gene therapy vectors such as adeno-associated virus (AAV), but current …
characterization of gene therapy vectors such as adeno-associated virus (AAV), but current …
Analytical methods to characterize recombinant adeno-associated virus vectors and the benefit of standardization and reference materials
EA Green, KH Lee - Current opinion in biotechnology, 2021 - Elsevier
Recombinant adeno-associated virus (rAAV) is an increasingly important gene therapy
vector, but its properties present unique challenges to critical quality attribute (CQA) …
vector, but its properties present unique challenges to critical quality attribute (CQA) …
A novel next-generation sequencing and analysis platform to assess the identity of recombinant adeno-associated viral preparations from viral DNA extracts
K Guerin, M Rego, D Bourges, I Ersing… - Human Gene …, 2020 - liebertpub.com
Recombinant adeno-associated virus (rAAV) vectors are increasingly popular gene delivery
tools in biological systems. They are safe and lead to high-level, long-term transgene …
tools in biological systems. They are safe and lead to high-level, long-term transgene …