# Improved Globin Expression Vectors for Gene Therapy of Human Hemoglobinopathies

> **NIH NIH F31** · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2020 · $48,279

## Abstract

PROJECT SUMMARY
A lentiviral vector (CCLc- βAS3-FB {βAS3LV}) is being investigated for the treatment of severe sickle cell
disease, however, it suffers from low titer, sub-optimal gene transfer to CD34+ hematopoietic stem cells
(HSCs), and expression likely insufficient to definitively cure β-thalassemia (although sufficient to prevent
sickling in pre-clinical studies). We hypothesize that there are known and unknown human β-globin genomic
sequences within βAS3LV that are inhibiting vector performance. Studies outlined in this proposal will
investigate how removal and/or addition of known or unknown elements within βAS3LV's human β-globin
genomic sequences affect titer, gene delivery to HSCs, and expression of the anti-sickling βAS3-globin gene.
The outcome of these studies will provide insight into how specific regulatory elements influence the
performance of βAS3LV across multiple categories. Moreover, this research will yield a second generation of
improved lentiviral vectors for efficiently transferring and effectively expressing the anti-sickling βAS3-globin
gene for gene therapy of sickle cell disease.

## Key facts

- **NIH application ID:** 9981803
- **Project number:** 5F31HL134313-05
- **Recipient organization:** UNIVERSITY OF CALIFORNIA LOS ANGELES
- **Principal Investigator:** Richard A Morgan
- **Activity code:** F31 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $48,279
- **Award type:** 5
- **Project period:** 2016-08-01 → 2021-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9981803

## Citation

> US National Institutes of Health, RePORTER application 9981803, Improved Globin Expression Vectors for Gene Therapy of Human Hemoglobinopathies (5F31HL134313-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9981803. Licensed CC0.

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