Scientific Publications

NCATS-supported scientists engage in cutting-edge research across the translational science spectrum. Explore the latest advances made possible by NCATS support.

Scientific Publications

Use the filter and search feature below to find recent scientific publications on NCATS-supported research.

Data Sharing to Advance Gene-targeted Therapies in Rare Diseases

Bespoke Gene Therapy Consortium (BGTC) Platform Vector Gene Therapy (PaVe-GT) - January 3, 2023 - American Journal of Medical Genetics, Seminars in Medical Genetics, Part C

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3-D Tissue Bioprinting
ASPIRE
Assay Guidance Manual (AGM)
Bespoke Gene Therapy Consortium (BGTC)
Biomedical Data Translator (Translator)
Clinical and Translational Science Awards (CTSA) Program
Extracellular RNA Communication (ExRNA)
Helping to End Addiction Long-term® Initiative (HEAL)
Illuminating Druggable Genome (IDG)
Informatics (IFX)
National Clinical Cohort Collaborative (N3C)
New Therapeutic Uses (NTU)
Platform Vector Gene Therapy (PaVe-GT)
Rare Diseases
Rare Diseases Clinical Research Network (RDCRN)
Small Business (SBIR)
Somatic Cell Genome Editing (SCGE)
Therapeutics for Rare and Neglected Diseases (TRND)
Tissue Chip for Drug Screening
Toxicology in the 21st Century (Tox21)
Translational Science Education

Scientific Publications

Scientific Publications

Data Sharing to Advance Gene-targeted Therapies in Rare Diseases

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