Rebecca Ahrens-Nicklas
Director, RASopathies Center · Children's Hospital of Philadelphia
Credentials: · · · · · · ·
History
- Present
RASopathies Center · Children's Hospital of Philadelphia
- Present
Division of Genetic and Genomic Medicine · Children's Hospital of Philadelphia
PresentUniversity of Pennsylvania Health System · Perelman School of Medicine at the University of Pennsylvania
- 2007–2011
2003
Research
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
Case report · New England Journal of Medicine · 2025 · 275 citations
Stimulation of entorhinal cortex-dentate gyrus circuitry is antidepressive
Nature Medicine · 2018 · 89 citations
Current clinical applications of AAV-mediated gene therapy
Review · Molecular Therapy · 2025 · 87 citations
17 papers
15 papers
13 papers
13 papers · 1 as a fellow
10 papers
8 papers
6 papers
6 papers
Grants
- 2024–2028
Personalized Prime Editing as a Platform for Hepatic Inborn Errors of Metabolism
National Center for Advancing Translational Sciences · U01
Contact PI · $10.8M · Children's Hosp of Philadelphia
- 2023–2028
Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
National Institute of Neurological Disorders and Stroke · U19
Project lead · $5.5M · Children's Hosp of Philadelphia
- 2023–2027
Network Modulation to Improve Gene Therapy in CLN3 Disease
National Institute of Neurological Disorders and Stroke · R01
Contact PI · $2M · Children's Hosp of Philadelphia
- 2022–2025
Disease Severity Stratification in Multiple Sulfatase Deficiency
National Institute of Child Health and Human Development · R21
Contact PI · $490.1K · Children's Hosp of Philadelphia
Clinical trials
- 2026–2028
Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders
Principal investigator · Recruiting · Phase 1, Phase 2
Rebecca Ahrens-Nicklas