Debugging the genetic code: non-viral in vivo delivery of therapeutic genome editing technologies.
Sign Up to like & getrecommendations! Published in 2018 at "Current opinion in biomedical engineering"
DOI: 10.1016/j.cobme.2018.08.002
Abstract: Efforts to precisely correct genomic mutations that underlie hereditary diseases for therapeutic benefit have advanced alongside the emergence and improvement of genome engineering technologies. These methods can be divided into two main classes: active nucleasebased… read more here.
Keywords: genome editing; non viral; delivery; vivo delivery ... See more keywords
Therapeutic Genome Editing in Cardiovascular Diseases
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DOI: 10.1016/j.jacbts.2018.11.004
Abstract: Summary A variety of genetic cardiovascular diseases may one day be curable using gene editing technology. Germline genome editing and correction promises to permanently remove monogenic cardiovascular disorders from the offspring and subsequent generations of… read more here.
Keywords: genome editing; variety; cardiovascular diseases; editing cardiovascular ... See more keywords
Therapeutic genome editing: regulatory horizons
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DOI: 10.1038/d41573-021-00130-7
Abstract: An increasing number of genome-editing technologies are being developed. Here, we highlight some of the key issues for future therapies using these technologies from a regulatory perspective. An increasing number of genome-editing technologies are being… read more here.
Keywords: genome editing; genome; regulatory horizons; editing regulatory ... See more keywords
The delivery challenge: fulfilling the promise of therapeutic genome editing
Sign Up to like & getrecommendations! Published in 2020 at "Nature Biotechnology"
DOI: 10.1038/s41587-020-0565-5
Abstract: Genome editing has the potential to treat an extensive range of incurable monogenic and complex diseases. In particular, advances in sequence-specific nuclease technologies have dramatically accelerated the development of therapeutic genome editing strategies that are… read more here.
Keywords: challenge fulfilling; genome editing; delivery challenge; genome ... See more keywords