Publications by authors named "S Felker"

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Genotype-First Assessment of Presentation and Penetrance of Neurofibromatosis Type 1, Autosomal Dominant Polycystic Kidney Disease, and Marfan Syndrome Within the All of Us Research Program Cohort.
Stephanie A Felker, Bruce R Korf, Gregory S Barsh

Genet Med· November 2025

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Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.
Michael Grimley, Stella M Davies, Archana Shrestha, Amy Shova, Monika Asnani, Sydney Felker

Nat Med· July 2025

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Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models.
Archana Shrestha, Devin M Pillis, Sydney Felker, Mengna Chi, Kimberly Wagner

Mol Ther Methods Clin Dev· June 2025

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A genotype-first approach identifies high incidence of NF1 pathogenic variants with distinct disease associations.
Anton Safonov, Tomoki T Nomakuchi, Elizabeth Chao, Carrie Horton, Jill S Dolinsky, Stephanie A Felker

Nat Commun· April 2025

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Genotype-First Assessment of Presentation and Penetrance of Neurofibromatosis Type 1, Autosomal Dominant Polycystic Kidney Disease, and Marfan Syndrome Within the Research Program Cohort.
Stephanie A Felker, Bruce R Korf, Gregory S Barsh

medRxiv· February 2025

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