# HSV VECTOR SYSTEMS FOR GENE THERAPY OF AGING DISORDERS

> **NIH NIH R01** · BOSTON CHILDREN'S HOSPITAL · 1992 · $167,207

## Abstract

Herpes Simplex Virus (HSV-1) vectors are being developed for gene therapy
of aging disorders such as Parkinson's Disease (PD).  The long range goal
of the project is to use HSV-1 vectors to deliver the genes responsible for
monamine synthesis directly into striatal neurons and glia, in the adult
mammalian brain, for human gene therapy of the aging disorder, PD.  Thus,
this project will also serve as a model for gene therapy approaches to
other aging disorders.  We have established that a HSV-1 vector expressing
human tyrosine hydroxylase (TH) can cause regulated release of both L-Dopa
and dopamine from cultured striatal neurons.  The overall goal of this
proposal is to use the striatal culture expression system, that is already
established, to characterize multiple vectors, in order to develop an
optimum vector for use in animal studies (Our colleagues are currently
performing parallel studies in the rat model of PD, not part of this
proposal.).  Among the parameters that will be examined are first, the
optimum striatal cell type for TH expression: This will be approached by
limiting TH expression to striatal neurons, or glia, or cholinergic
neurons, by constructing and characterizing vectors that express TH from
the i) human neurofilament L, ii) rat GFAP, and iii) rat choline acetyl
transferase promoters.  Second, we will construct and characterize vectors
that coexpress TH and aromatic amino acid decarboxylase (ADDC) in the same
cell, to determine if coexpression of TH and AADC causes efficient
production of dopamine.  Lastly, we will improve the defective HSV-L vector
packaging system to support use in humans.

## Key facts

- **NIH application ID:** 3122789
- **Project number:** 1R01AG010827-01
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **SAM.gov UEI:** Z1L9F1MM1RY3
- **Principal Investigator:** ALFRED I. GELLER
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 1992
- **Award amount:** $167,207
- **Award type:** 1
- **Project period:** 1992-09-30 → 1995-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/3122789

## Citation

> US National Institutes of Health, RePORTER application 3122789, HSV VECTOR SYSTEMS FOR GENE THERAPY OF AGING DISORDERS (1R01AG010827-01). Retrieved via MORVS 2026-10-25 from https://api.morvs.ai/grant/nih/3122789. Licensed CC0.

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