# Beginning genetic interventions in infants (BEGIN)

> **NIH HG R01** · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · 2026 · $3,151,302

## Abstract

PROJECT SUMMARY
The advancements in genomic medicine have significantly accelerated over the past decade. However, their
translation into clinical practice have been slow, and the precise treatment options are essentially nonexistent.
The formulation of effective therapies is obstructed by the vast diversity of genetic diseases and the limited
number of patients diagnosed with any specific genetic condition. Furthermore, the inefficiency often
encountered in diagnostic workups leads to delayed diagnoses for children, rendering it difficult to test
interventions effectively at an appropriate stage of their condition. There exists an urgent necessity to bridge
the disparities between the prompt identification of patients who are likely to have a genetic disease, the
delineation of a precise genetic diagnosis, and the development of effective precision therapies.
This project is centered on the Neonatal Intensive Care Unit (NICU) and establishes a framework for an
innovative approach to addressing its challenges by integrating rapid turnaround diagnostics with expedited
gene-targeted therapies. Within this model, we will provide rapid genome sequencing to patients in the NICU,
where many severe childhood genetic conditions initially present—approximately 20% of all admissions—thus
offering the opportunity for early diagnosis, prior to irreversible disease progression. Furthermore, we will
leverage our VIGOR network and collaborate with comprehensive sequencing facilities, such as GeneDx, to
identify NICU cases that exhibit specific types of mutations and conditions that qualify them for patient-
customized antisense oligonucleotides (ASOs) therapies. These ASOs are modular therapeutic agents
composed of snippets of synthetic DNA or RNA, ranging from 15-30 nucleotides, which can be flexibly tailored
to modulate specific gene-splicing patterns or target genes for degradation. Our previous work has
demonstrated the feasibility of developing ASOs as a platform for precisio

## Key facts

- **NIH application ID:** 11291997
- **Project number:** 1R01HG014337-01A1
- **Recipient organization:** UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
- **Principal Investigator:** PANKAJ B AGRAWAL; Timothy Wei-Wen Yu
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** HG
- **Fiscal year:** 2026
- **Award amount:** $3,151,302
- **Award type:** 1
- **Project period:** 2026-04-03T00:00:00 → 2030-03-31T00:00:00

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11291997

## Citation

> US National Institutes of Health, RePORTER application 11291997, Beginning genetic interventions in infants (BEGIN) (1R01HG014337-01A1). Retrieved via AI Analytics 2026-05-18 from https://api.ai-analytics.org/grant/nih/11291997. Licensed CC0.

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