Timothy W. Yu

机构：Broad Institute, Boston Children's Hospital, Howard Hughes Medical Institute, Harvard University, Somerville Hospital, Cambridge Collaborative (United States), Dana-Farber/Boston Children's Cancer and Blood Disorders Center · ORCID：0000-0003-2988-7701

发表论文 184 篇 · 总被引 25046 次 · h-index 58

代表论文

• Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments (2025 · The American Journal of Human Genetics · 被引 24)
• The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing (2025 · Nucleic Acids Research · 被引 17)
• The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK (2025 · Nature Medicine · 被引 8)
• From roadmap to a sustainable end-to-end individualized therapy pathway (2025 · Therapeutic Advances in Rare Disease · 被引 7)
• Multi-omic analysis of the ciliogenic transcription factor RFX3 reveals a role in promoting activity-dependent responses via enhancing CREB binding in human neurons (2025 · bioRxiv (Cold Spring Harbor Laboratory) · 被引 6)
• Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy (2026 · Nature Medicine · 被引 4)