Award
National Human Genome Research Institute 1R01HG014337-01A1
Beginning genetic interventions in infants (BEGIN)
Recipient
University of Miami School of Medicine, Coral Gables, FL, United States
Award Amount
$3,151,302.00
Ceiling
$3,151,302.00
Awarded
April 07, 2026
Identifier
1R01HG014337-01A1
This award funds a project to implement precision medicine in the NICU by integrating rapid genome sequencing and gene-targeted ASO therapies to treat infants with severe genetic disorders early, aiming to transform neonatal care.
Description
The project aims to develop a clinical framework for early intervention precision medicine in the Neonatal Intensive Care Unit (NICU). It focuses on addressing severe genetic diseases in infants through rapid genome sequencing and antisense oligonucleotide (ASO) therapies. The initiative seeks to bridge the gap between genetic diagnosis and effective therapies, enabling early diagnosis and personalized treatment for infants with genetic conditions.