# Combination gene editing protocol using CRISPR technology for HIV/AIDS cure in non-human primates

> **NIH NIH R56** · TEMPLE UNIV OF THE COMMONWEALTH · 2020 · $824,124

## Abstract

Even
increased
the
with contemporary anti-retroviral therapy (ART) regimens that
the survival rate , HIV remain s an enormous health burden
disease by eliminating proviral DNA, which is present in the genome of infected host cells.
suppress
viral
. Unfortunately, ART has
replication and have
failed to cure
The persistence
of the viral genome in tissues with the potential to become reactivated, when ART is discontinued, underscores
the importance of alternative, perhaps companion, strategies for eradication of the HIV genome from the
latently infected reservoirs. The possibility of a curative treatment for HIV infection has been energized by the
“Berlin patient” and more recently the “London patient”, who both received stem cell transplants from a
CCR532-homozygous donor after radiation and chemotherapy for leukemia and have been undetectable for
the virus in his blood for 12 and 2 years since stopping ART after the transplants. CCR5 is a cellular protein
that serves a co-receptor for HIV infection, therefore its genetic inactivation prevents viral infection and its
spread throughout the body of the infected individual. In previous studies, we successfully applied CRISPR-
Cas9 gene editing strategy to target and eradicate integrated HIV sequences in in vitro cell culture models, ex
vivo cultured HIV+ patient derived and in vivo in HIV-infected humanized mice model. We presented data at
CROI 2019 and here of in vivo editing of the SIV genome in blood of SIV-infected macaques after i.v. injection
of AAV9/CRISPR-Cas9. In this multi Principal investigator (mPi) grant application, we will test the hypothesis
that employment of a combined gene editing platform can effectively excise SIV proviral DNA and further
editing of the CCR5 gene will remove viral targets in the animal leading to a reduction in the functional viral
reservoir and/or complete elimination of replication competent virus in the treated animals. To this end, we will
employ CRISPR-Cas9 technology for sequential targeting and permanent inactivation of both SIV and CCR5 in
an SIV-infected non-human primate model. To achieve our goals, we
investigators
have formed a team of experienced
with extensive experience in in vivo SIV animal models and immunology (T. Burdo, mPi, Temple
University), gene editing technologies for eradicating virus from host genomes in in vitro cell cultures and in
vivo animal models
novel assays In this application, we will establish the CCR5
and dual SIV and CCR5 gene editing platforms in SIV-infected non-human primates and determine the
immunological and virological effects and the underlying mechanism of cure or delayed viral rebound after
gene editing and antiretroviral therapy interruption (ATI). We will use single and then a sequential dual editing
targeting the cellular gene (CCR5) followed by the SIV proviral DNA. The outcome of these studies will offer
valuable insight into the potential of CRISPR-Cas9 gene editing for consideration in the HIV cure strategie...

## Key facts

- **NIH application ID:** 10153532
- **Project number:** 1R56AI150772-01A1
- **Recipient organization:** TEMPLE UNIV OF THE COMMONWEALTH
- **Principal Investigator:** Tricia Helen Burdo
- **Activity code:** R56 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $824,124
- **Award type:** 1
- **Project period:** 2020-07-01 → 2023-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10153532

## Citation

> US National Institutes of Health, RePORTER application 10153532, Combination gene editing protocol using CRISPR technology for HIV/AIDS cure in non-human primates (1R56AI150772-01A1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10153532. Licensed CC0.

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