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First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Crossref DOI link: https://doi.org/10.1016/j.ymthe.2022.10.010

Published Print: 2022-12

Update policy: https://doi.org/10.1016/elsevier_cm_policy

Authors

Harmatz, Paul

Prada, Carlos E.

Burton, Barbara K.

Lau, Heather

Kessler, Craig M.

Cao, Liching

Falaleeva, Marina

Villegas, Andres G.

Zeitler, Jennifer

Meyer, Kathleen

Miller, Weston

Wong Po Foo, Cheryl

Vaidya, Sagar

Swenson, Wendy

Shiue, Lisa H.

Rouy, Didier

Muenzer, Joseph
Funding

Funding for this research was provided by:

Sangamo Therapeutics
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Text and Data Mining valid from 2022-12-01

Text and Data Mining valid from 2022-12-01

Version of Record valid from 2022-10-26
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This article is maintained by: Elsevier

Article Title: First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Journal Title: Molecular Therapy

CrossRef DOI link to publisher maintained version: https://doi.org/10.1016/j.ymthe.2022.10.010

Content Type: article

Copyright: © 2022 The Authors.