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 …
[HTML][HTML] 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 …
MicroRNA regulation of atherosclerosis
MW Feinberg, KJ Moore - Circulation research, 2016 - Am Heart Assoc
Atherosclerosis and its attendant clinical complications, such as myocardial infarction,
stroke, and peripheral artery disease, are the leading cause of morbidity and mortality in …
stroke, and peripheral artery disease, are the leading cause of morbidity and mortality in …
Non-coding RNAs in cardiovascular diseases: diagnostic and therapeutic perspectives
Recent research has demonstrated that the non-coding genome plays a key role in genetic
programming and gene regulation during development as well as in health and …
programming and gene regulation during development as well as in health and …
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 …
Adeno-associated virus-based gene therapy for CNS diseases
M Hocquemiller, L Giersch, M Audrain… - Human gene …, 2016 - liebertpub.com
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …
providing a durable therapeutic protein via a single administration. Adeno-associated virus …
[HTML][HTML] Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates
Delivering genes to and across the brain vasculature efficiently and specifically across
species remains a critical challenge for addressing neurological diseases. We have evolved …
species remains a critical challenge for addressing neurological diseases. We have evolved …
[HTML][HTML] Fantastic AAV gene therapy vectors and how to find them—random diversification, rational design and machine learning
Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …
variety of species, tissues and cell types. For over half a century, their intriguing biology and …
Next-generation AAV vectors—do not judge a virus (only) by its cover
C Domenger, D Grimm - Human molecular genetics, 2019 - academic.oup.com
Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …
numerous clinical trials after they have emerged as a highly promising vector for human …
[HTML][HTML] Adeno-associated viruses (AAV) and host immunity–a race between the hare and the hedgehog
K Rapti, D Grimm - Frontiers in immunology, 2021 - frontiersin.org
Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …
the basis for several approved gene therapies for human diseases, mainly owing to their …