# CRISPR for Cure

> **NIH NIH UM1** · TEMPLE UNIV OF THE COMMONWEALTH · 2024 · $4,807,696

## Abstract

While antiretroviral therapy (ART) has dramatically reduced HIV disease morbidity and mortality, it has failed to
eliminate viral reservoirs. Interruption of treatment leads to activation of latent virus and rebound viremia within
weeks. Novel strategies are urgently needed to eradicate latent infections and enhance the immune system
leading to sustained, durable control of viral rebound following the cessation of ART. In response to RFA-AI-
20-035 Martin Delaney Collaboratories for HIV Cure Research, we now submit the application entitled
"CRISPR for Cure." The overarching goal of this program is to use genome editing mediated by CRISPR to
enhance immune responses and directly ablate HIV proviruses. We have assembled a collaborative team of
highly accomplished basic and translational scientists working in tandem with community stakeholders and a
small biotechnology company to develop CRISPR-based therapies to directly target the HIV provirus and to
enhance immunological responses. The research program is comprised of three highly interactive research
foci (RF) that will utilize interdisciplinary, innovative and collaborative research approaches with community
and government input. RF1 will use next generation sequencing and novel barcoded viruses to define the HIV
reservoir and the impact of epigenetic mechanisms on proviral rebound. In RF2, we will enhance effector NK
and CTL cell function and killing and limit viral spread by target cells using innovative genome editing
strategies. RF3 will create and test the next generation of inducible, multiplex CRISPR with increased
specificity, potency and safety for delivery by CD4 tropic lymphoid AAV9 for eradication of HIV-1 proviral DNA
in animal models whose immune cells are modified in RF2 and assess the possibility of both a universal and
personalized CRISPR in eliminating replication competent virus in vivo. In addition to the shared focus on
CRISPRs technology, the Collaboratory will undertake a highly integrated experimental agenda through the
shared use of barcoded viruses in humanized mice and unique
support
MISTRG humanized mice that differentially
human hematopoietic stem and progenitor cell maintenance and myelopoiesis;rhesus macaques
infected with a novel SIV barcoded virus; ex vivo clinical samples from a well characterized cohort and the use
of adenoviruses to efficiently deliver CRISPRs to an in vivo humanized animal model carrying cells from
patient-derived PBMCs. The outcome of this comprehensive and multidisciplinary program by the “CRISPR for
Cure”
the
Collaboratory, will accelerate the use of gene editing strategies towards eradication of HIV infection from
body or sustained viral remission following cessation of antiretroviral therapy.With resources available
from our private sector partner, we will be well positioned for further GMP manufacturing development, and
future initial clinical investigations.

## Key facts

- **NIH application ID:** 10864023
- **Project number:** 5UM1AI164568-04
- **Recipient organization:** TEMPLE UNIV OF THE COMMONWEALTH
- **Principal Investigator:** Tricia Helen Burdo
- **Activity code:** UM1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $4,807,696
- **Award type:** 5
- **Project period:** 2021-08-16 → 2026-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10864023

## Citation

> US National Institutes of Health, RePORTER application 10864023, CRISPR for Cure (5UM1AI164568-04). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10864023. Licensed CC0.

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