The clinical progress of mRNA vaccines and immunotherapies
The emergency use authorizations (EUAs) of two mRNA-based severe acute respiratory
syndrome coronavirus (SARS-CoV)-2 vaccines approximately 11 months after publication of …
syndrome coronavirus (SARS-CoV)-2 vaccines approximately 11 months after publication of …
AAV vectors: The Rubik's cube of human gene therapy
A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration
JA Greig, KM Martins, C Breton, RJ Lamontagne… - Nature …, 2024 - nature.com
The development of liver-based adeno-associated virus (AAV) gene therapies is facing
concerns about limited efficiency and durability of transgene expression. We evaluated …
concerns about limited efficiency and durability of transgene expression. We evaluated …
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 …
Immune responses to viral gene therapy vectors
JL Shirley, YP de Jong, C Terhorst, RW Herzog - Molecular Therapy, 2020 - cell.com
Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …
much larger number of additional viral vectors are in various stages of clinical trials for the …
Adeno-associated virus vector as a platform for gene therapy delivery
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …
treatment of a variety of human diseases. Recent advances in developing clinically …
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 …
In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments
such as gene therapy or bispecific antibody therapy have been introduced, substantial …
such as gene therapy or bispecific antibody therapy have been introduced, substantial …
CRISPR-Cas9 DNA base-editing and prime-editing
Many genetic diseases and undesirable traits are due to base-pair alterations in genomic
DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic …
DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic …
Comprehensive assessment of miniature CRISPR-Cas12f nucleases for gene disruption
Because of their small size, the recently developed CRISPR-Cas12f nucleases can be
effectively packaged into adeno-associated viruses for gene therapy. However, a systematic …
effectively packaged into adeno-associated viruses for gene therapy. However, a systematic …