[HTML][HTML] Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
[HTML][HTML] Immunogenicity and toxicity of AAV gene therapy
HCJ Ertl - Frontiers in Immunology, 2022 - frontiersin.org
Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …
[HTML][HTML] Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
KA Strauss, MA Farrar, F Muntoni, K Saito… - Nature medicine, 2022 - nature.com
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …
Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice
The most common form of genetic heart disease is hypertrophic cardiomyopathy (HCM),
which is caused by variants in cardiac sarcomeric genes and leads to abnormal heart …
which is caused by variants in cardiac sarcomeric genes and leads to abnormal heart …
[HTML][HTML] Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors
The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
[HTML][HTML] Gene therapy advances: a meta-analysis of AAV usage in clinical settings
HKE Au, M Isalan, M Mielcarek - Frontiers in medicine, 2022 - frontiersin.org
Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …
drive long-term transgene expression in gene therapy. While animal studies have shown …
[HTML][HTML] Evaluating the state of the science for adeno-associated virus integration: an integrated perspective
DE Sabatino, FD Bushman, RJ Chandler, RG Crystal… - Molecular Therapy, 2022 - cell.com
On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …
Viral vector‐based gene therapies in the clinic
Gene therapies are currently one of the most investigated therapeutic modalities in both the
preclinical and clinical settings and have shown promise in treating a diverse spectrum of …
preclinical and clinical settings and have shown promise in treating a diverse spectrum of …
[HTML][HTML] rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …
[HTML][HTML] Addressing high dose AAV toxicity–'one and done'or 'slower and lower'?
TK Kishimoto, RJ Samulski - Expert opinion on biological therapy, 2022 - Taylor & Francis
While there has been substantial progress and notable achievements in the use of adeno-
associated virus (AAV) gene therapy vectors for treatment of rare diseases, setbacks related …
associated virus (AAV) gene therapy vectors for treatment of rare diseases, setbacks related …