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NOBEL PRIZE 2026

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NIHNATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OFF-TARGET RESOURCE CORE

Active
Institution
CHILDREN'S HOSP OF PHILADELPHIA, PA
Principal investigator
Xiao Wang
Funding
$712K
Duration
2023–2028
Open portfolio

Moonbase score

Still being scored

Abstract

PROJECT SUMMARY Off-target editing analyses will be so fundamental to the development of our therapeutic leads, regardless of the disease treated—whether phenylketonuria (Lead Project 1), hereditary tyrosinemia type 1 (Project 2), or mucopolysaccharidosis type I (Project 3)—and so integral to any IND applications that ultimately emerge from the Projects, that we are proposing an Off-Target Resource Core that will serve all three Research Projects. This Resource Core will provide services that are directly aligned with the preclinical studies that are recommended by the U.S. Food and Drug Administration (FDA) Draft Guidance for Industry on Human Gene Therapy Products Incorporating Human Genome Editing, namely: (1) identification of on-target and off-target editing activity, including the type, frequency, and location of all off-target editing events, taking into account human genetic variation; (2) assessment of genomic integrity, including chromosomal rearrangements, large insertions, or large deletions; and (3) evaluation of the biological consequences associated with off-target editing.

LOADING MOONBASE SCORE

NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKEU19Special Emphasis Panel[ZRG1-MGG-G]resourceeditingdraftinsertionsoff-targettaking accounton-targethuman gene

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