# Viral Vector Core

> **NIH NIH P01** · UNIV OF MASSACHUSETTS MED SCH WORCESTER · 2020 · $224,850

## Abstract

Project Summary – Core B: Vector Core
Recombinant adeno-associated virus (rAAV) is a popular gene transfer platform technology to deliver
transgenes for in vivo gene therapy. Our recent discovery of a diverse family of novel primate AAVs that are
capable of transducing a variety of target tissues much more robustly than the first generation of AAV2-based
vectors remarkably expanded the utility of rAAVs in different gene therapy applications including treatment of
alpha-1 antitrypsin deficiency (AATD). The Viral Vector Core (Core B) is the central component of our
collaborative effort to develop new approaches to gene therapy for AATD. The main objectives of Core B are to
provide the investigators of other subprojects and Cores with high quality of preclinical grade rAAV vectors to
address their specific scientific questions in a variety of animal models as well as reliable and reproducible
AAV neutralizing antibody testing service. Core B will rely on the resources and experience of the University of
Massachusetts Viral Vector Core (Guangping Gao, Director), an internationally well recognized vector service
with abundant experience in vector production, quality control, and uses in different in vivo gene transfer
applications to accomplish its goal through the following Aims: Aim 1. To design, create, produce, purify and
quality control test preclinical grade rAAV vector preparations for all subprojects and cores of this tPPG, and
Aim 2. To perform in vitro and in vivo neutralizing antibody assays to screen nonhuman primate animals for
studies proposed by subproject 2 and study seroepidemiology of novel AAV capsids developed by subproject 4
in human populations.

## Key facts

- **NIH application ID:** 9935126
- **Project number:** 5P01HL131471-05
- **Recipient organization:** UNIV OF MASSACHUSETTS MED SCH WORCESTER
- **Principal Investigator:** Guangping Gao
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $224,850
- **Award type:** 5
- **Project period:** — → 2022-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9935126

## Citation

> US National Institutes of Health, RePORTER application 9935126, Viral Vector Core (5P01HL131471-05). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9935126. Licensed CC0.

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