Genetic disorders
2 papers
Trial sponsors
Company9 trialsPhase 4498 people
Memorial Sloan Kettering Cancer Center
1 trialPhase 225 people
1 trialPhase 214 people
Funding
1 grantNational Heart, Lung, and Blood InstituteUS$7.7m
Latest
12 in 12 monthsPreprints.org
Additive Gene and Stem Cell Therapy for Mutation-Independent Treatment of p63 Disorders
Preprint: experiments in human cellsInterventions
Advanced Healthcare Materials
Ultrasound-Mediated CRISPR Delivery and Sonogenetic Modulation for Precision Therapeutics
ReviewInterventions
Journal of Clinical Lipidology
Familial hypercholesterolemia cascade testing: Insights from the Dutch Program
Cross-sectional study of 31,487 peopleFamilial hypercholesterolemia
Cell Proliferation
Liver Organoids: From Disease Modelling to Regenerative Medicine
ReviewInterventions
Aging Cell
Targeting RANKL Prevents Bone Loss, Improves Muscle Function and Extends Lifespan in Progeroid Mice
Experiments in miceMuscle
Ageing Research Reviews
Testicular aging revisited: Current understanding of mechanisms and emerging interventions
ReviewMechanisms
Advanced Science
First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria
Experiments in miceProgeria
Trends in Molecular Medicine
Neuroendocrine timekeepers: changes in normal and premature aging
ReviewMechanisms