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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 …
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 …
Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors
CRISPR–Cas9 paired with adeno-associated virus serotype 6 (AAV6) is among the most
efficient tools for producing targeted gene knockins. Here, we report that this system can …
efficient tools for producing targeted gene knockins. Here, we report that this system can …
Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): where are we, and how did we get here?
The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …
in Europe and the United States are landmark achievements in the history of modern …
AAV-mediated gene therapy for research and therapeutic purposes
RJ Samulski, N Muzyczka - Annual review of virology, 2014 - annualreviews.org
Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …
AAV cis-regulatory sequences are correlated with ocular toxicity
Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their
many advantages, including their reduced inflammatory profile compared with that of other …
many advantages, including their reduced inflammatory profile compared with that of other …
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
Recombinant adeno-associated viral vectors (AAVs) are an effective system for gene
transfer. AAV serotype 2 (AAV2) is commonly used to deliver transgenes to retinal ganglion …
transfer. AAV serotype 2 (AAV2) is commonly used to deliver transgenes to retinal ganglion …
Cystic fibrosis: genotypic and phenotypic variations
J Zielenski, LC Tsui - Annual review of genetics, 1995 - go.gale.com
Cystic fibrosis is a recessive disorder common among Caucasians. Although the disease
has been known in the 1930s, it was only in the late 1980s that the molecular mechanisms …
has been known in the 1930s, it was only in the late 1980s that the molecular mechanisms …
Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity
JE Rabinowitz, F Rolling, C Li, H Conrath… - Journal of …, 2002 - journals.asm.org
The serotypes of adeno-associated virus (AAV) have the potential to become important
resources for clinical gene therapy. In an effort to compare the role of serotype-specific virion …
resources for clinical gene therapy. In an effort to compare the role of serotype-specific virion …
Integration of adeno-associated virus (AAV) and recombinant AAV vectors
▪ Abstract The driving interest in adeno-associated virus (AAV) has been its potential as a
gene delivery vector. The early observation that AAV can establish a latent infection by …
gene delivery vector. The early observation that AAV can establish a latent infection by …