‘Safe harbor’ gene therapy approach may have first success

An obscure gene editor was used to restore a missing liver enzyme in an infant with a devastating metabolic condition.

Authors

Publication Details

Journal
Science
Published
2025-01-16
DOI
https://doi.org/10.1126/science.adv9833
Citations
1
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.81
Controls
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article

‘Safe harbor’ gene therapy approach may have first success

Jocelyn Kaiser
1 citations
Science
CRISPR and Genetic Engineering
0.81
article

‘Safe harbor’ gene therapy approach may have first success

Jocelyn Kaiser
article en
1 citations

Abstract

An obscure gene editor was used to restore a missing liver enzyme in an infant with a devastating metabolic condition.

ScienceVol. 387(6731)
Openalex Percentile: Top 32%
CRISPR and Genetic Engineering
0.81
DOISourceOpenAlex
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‘Safe harbor’ gene therapy approach may have first success — Jocelyn Kaiser · Science (2025) | TGRS Research Map | TGRS