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Hereditary Disease — NIH Grants & Funding

Active and recent NIH-funded research on Hereditary Disease. Data from NIH RePORTER, last 36 months.

MONDO:0003847

NIH-funded research on Hereditary Disease

active grants

institutions

researchers

new in last 12 mo.

$65.6M in NIH funding over the last 36 months. Data from NIH RePORTER.

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Basic Science(33)

Basic ScienceR01Active

Activating Autophagy to Treat Uromodulin-Associated Genetic Chronic Kidney Disease

Chen, Ying·WASHINGTON UNIVERSITY·SAINT LOUIS, MO

$2,969,015 (FY2025)

Hereditary Disease — NIH Grants & Funding

Basic Science(33)

Activating Autophagy to Treat Uromodulin-Associated Genetic Chronic Kidney Disease

Pathobiology and treatment of patients with inborn errors of immunity

DNA repair

Inherited Ocular Diseases

Assessing therapeutic intervention of short telomere syndromes

Investigation of the genetic cause of inherited disorders of reproduction

Proteins From Hereditary Eye Diseases: In silico and Experimental Studies

Mechanisms of Neurodegeneration in Hereditary Prion Diseases

Imaging Assessments of ARPKD Kidney Disease Progression

Investigating the Function of Highly Similar Intrachromosomal Repeats to Genomic Instability and Perturbed Gene Expression in Genetic Disorder

Translational(5)

Skin probiotics to treat Netherton Syndrome

Developing hemophilia A therapeutics by targeting translational and posttranslational regulation of FVIII

Innovative Approaches to Treating Alport Syndrome

MiR-155 and RUNX function in neurofibroma tumorigenesis and therapy

Identifying therapeutic strategies for the multisystem genetic disorder Pseudoxanthoma Elasticum

Clinical(9)

Phase IA/IB Study of AAVrh.10hFXN Therapy to Treat the Cardiomyopathy of Friedreich's Ataxia

Platelet function abnormalities underlying bleeding in a connective tissue disorder the Ehlers Danlos syndrome

Genetic Modifiers of Retinal Disease

Improving intestinal symptoms in a Congenital Disorder of Glycosylation

Daily Caloric Restriction in Overweight and Obese Adults with ADPKD

Mechanisms of calcium-induced arrhythmias in arrhythmogenic right ventricular cardiomyopathy

Evaluating causes for failure to thrive in a Noonan-like syndrome with loose anagen hair (NSLH) patients using NSLH vertebrate model

Prospective Studies Of Phlebotomy Therapy In Hereditary Hemochromatosis

Hereditary Cancer Variants of Uncertain Significance in Stem Cells

Epi / Nat. History(2)

Delineation of the natural history of Ollier disease and Muffucci syndrome and investigation of their genetic bases

Natural History of Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD), a Heritable Disorder of GABA Metabolism

Gene/Cell Therapy(18)

Gene Therapy to Treat Ethanol-induced Osteoporosis Associated with Aldehyde Dehydrogenase 2 Deficiency in Post-menopausal East Asian Women

Nonhuman Primate Model of Inherited Photoreceptor Degeneration

Development of a long-lasting, bone-targeted gene therapy for osteogenesis imperfecta

Allele Specific Knockdown for LGMDD1

Intracerebroventricular cyclodextrin and AAV-mediated gene therapy forglobal CNS disease correction in feline NPC1 disease

First murine animal model and adeno-associated virus (AAV)-based gene therapy for MTATP6 mitochondrial diseases

A one and done therapeutic strategy to correct ELP1 splicing defect in familial dysautonomia

Anticodon Engineered tRNA for the Treatment of Hereditary Eye Disease

Precision Gene Editing on Osteogenesis Imperfecta

Developing strategies to identify candidate individuals for early genetic therapies

Biomarker / Dx(3)

Biomarkers for treatment monitoring and prognostication of disorders of hepatic copper metabolism

Extracellular RNA biomarkers of myotonic dystrophy type 1

Expert curation of clinically significant variants in genes for early onset retinal degeneration

Other(4)

Clinical Genetic Studies of Familial and Hereditary Cancer Syndromes

Delivering inhalable nanoparticles containing AI-optimized CFTR mRNA with a novel microfluidic device for treatment of CF

Multimodal machine learning for diagnosis and mechanistic phenotyping of inherited diseases

Delineating pathogenic effects of ATP2C1 loss-of-function in human keratinocytes and organotypic epidermis to identify therapeutic strategies for Hailey-Hailey disease