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 …
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 …
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 …
Progress and problems with the use of viral vectors for gene therapy
CE Thomas, A Ehrhardt, MA Kay - Nature Reviews Genetics, 2003 - nature.com
Gene therapy has a history of controversy. Encouraging results are starting to emerge from
the clinic, but questions are still being asked about the safety of this new molecular …
the clinic, but questions are still being asked about the safety of this new molecular …
Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy
A Srivastava - Molecular Therapy-Nucleic Acids, 2023 - cell.com
Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy
Attempts to develop gene therapy for Duchenne muscular dystrophy (DMD) have been
complicated by the enormous size of the dystrophin gene. We have performed a detailed …
complicated by the enormous size of the dystrophin gene. We have performed a detailed …
Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps
JC Grieger, RJ Samulski - Journal of virology, 2005 - Am Soc Microbiol
The limited packaging capacity of adeno-associated virus (AAV) precludes the design of
vectors for the treatment of diseases associated with larger genes. Autonomous …
vectors for the treatment of diseases associated with larger genes. Autonomous …
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
B Wang, J Li, X Xiao - … of the National Academy of Sciences, 2000 - National Acad Sciences
Duchenne muscular dystrophy (DMD) is the most common and lethal genetic muscle
disorder, caused by recessive mutations in the dystrophin gene. One of every 3,500 males …
disorder, caused by recessive mutations in the dystrophin gene. One of every 3,500 males …
Viral vectors for gene delivery to the central nervous system
TB Lentz, SJ Gray, RJ Samulski - Neurobiology of disease, 2012 - Elsevier
The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …