# PedGeneRx - Admin Supplement to Base Editing and Prime Editing for Sickle Cell Disease R01

> **NIH NIH R01** · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · 2022 · $182,000

## Abstract

ABSTRACT:
There is a substantial unmet need for the development of new treatments for patients with monogenic disorders
such as sickle cell disease (SCD) and gene therapy technologies represent a potential novel solution. This
project addresses an important issue in research ethics – improving the quality of informed consent and building
capacity for gene therapy communication with a vulnerable population of research participants (pediatric
patients, as well as underrepresented minorities). Since early treatment can reduce long-term morbidity, improve
quality of life, and reduce early mortality it is imperative to focus on pediatric patients. In considering gene
therapy, families face complex treatment choices when considering whether they should pursue a novel therapy
where the long-term effects are uncertain, and the risk-benefit ratios of the different treatment options are difficult
to compare. We hypothesize that families have unique informational needs surrounding these treatments and
dedicated educational platforms with decisional aids will be crucial to promoting collaborative, family-centered
decision-making and overall satisfaction when considering gene therapy for their child's rare disease. Under this
administrative supplement, we will conduct a needs assessment (Aim 1) that includes approximately 50 semi-
structured interviews with parents whose child (a) had (or been offered) gene therapy or (b) has a disorder where
pediatric gene therapy trails are on the horizon. To complete a truly comprehensive needs assessment of the
gaps and wants around gene therapy, we will interview 10-20 health care providers with experience caring for
pediatric gene therapy families. We will then develop and refine a multimedia web platform that provides families
with a toolkit of information about gene therapy, which will include a pediatric gene therapy support tool we
develop as part of this aim (Aim 2). To achieve the aims of this patient-focus quality improvement project: the
interviews will be audio recorded, transcribed verbatim, and analyzed using semantic-content analysis by an
expert team of mixed-methods researchers. We will use validated tools and follow the international standards
for patient decision aids. The tools will be developed and refined iteratively through well-established methods of
alpha and beta testing pilot content. Our research team plans to work with Booster Shot Media, a company
experienced in creating patient-centered content for researchers and academic medical centers to communicate
about other aspects of healthcare. This project is innovative and will fill an important gap in patient-provider
communication around gene therapy for pediatric disease. Once finalized, this will be the first (to our knowledge)
validated, evidenced based decisional-support toolkit available to parents of children with rare disease who are
eligible for gene therapy.

## Key facts

- **NIH application ID:** 10594247
- **Project number:** 3R01HL156647-02S1
- **Recipient organization:** ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- **Principal Investigator:** DAVID R LIU
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $182,000
- **Award type:** 3
- **Project period:** 2021-01-01 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10594247

## Citation

> US National Institutes of Health, RePORTER application 10594247, PedGeneRx - Admin Supplement to Base Editing and Prime Editing for Sickle Cell Disease R01 (3R01HL156647-02S1). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/10594247. Licensed CC0.

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