[HTML][HTML] 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 …
[HTML][HTML] 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 …
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 …
Base editing: precision chemistry on the genome and transcriptome of living cells
RNA-guided programmable nucleases from CRISPR systems generate precise breaks in
DNA or RNA at specified positions. In cells, this activity can lead to changes in DNA …
DNA or RNA at specified positions. In cells, this activity can lead to changes in DNA …
Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases
H Jang, DH Jo, CS Cho, JH Shin, JH Seo… - Nature biomedical …, 2022 - nature.com
The use of prime editing—a gene-editing technique that induces small genetic changes
without the need for donor DNA and without causing double strand breaks—to correct …
without the need for donor DNA and without causing double strand breaks—to correct …
Deafness: From genetic architecture to gene therapy
Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Adenine base editors (ABEs) composed of an engineered adenine deaminase and the
Streptococcus pyogenes Cas9 nickase enable adenine-to-guanine (A-to-G) single …
Streptococcus pyogenes Cas9 nickase enable adenine-to-guanine (A-to-G) single …
[PDF][PDF] CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
CE Nelson, CH Hakim, DG Ousterout, PI Thakore… - Science, 2016 - science.org
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000
male births and caused by mutations in the dystrophin gene. Genome editing has the …
male births and caused by mutations in the dystrophin gene. Genome editing has the …
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …