# ACTIVATION OF IMMUNOCOMPETENT LYMPHOCYTES

> **NIH NIH R01** · NEW YORK UNIVERSITY · 1988 · $252,928

## Abstract

Until recently, primary immunodeficiency disorders in man have been classified
as to whether they appear to involve cellular immunity, humoral immunity, or
both together.  However, many disorders do not fit easily into such
classification.  The discovery of specific inherited molecular defects which
give rise to immunodeficiency syndromes has provided an alternative approach.
Further elucidation of specific molecular defects, as well as of the genetic
heterogeneity within each of the known defects, would provide for a clearer
understanding of abnormal immune function.  Elucidation of the pathogenetic
mechanisms involved in the development of immunodeficiency may provide tools for
the metabolic manipulation of the normal and abnormal immune system.  Three
specific (adenosine deaminase, purine nucleoside phosphorylase and
transcobalamin II deficiency) and possibly a fourth (5'-nucleoside deficiency)
have been described.  Three of these defects involve the purine metabolic
pathway (crucial for synthesis of the substrates for DNA synthesis) and result
in accumulation of potentially toxic metabolites such as cAMP, dATP and dGTP.
We will therefore determine the extent of genetic heterogeneity in ADA
deficiency.  We will develop in vitro and in vivo models of immune function to
define the consequences of deficiency of ADA and PNP and test the effectiveness
of enzyme replacement versus other metabolic manipulations in reversing these
consequences.  We will characterize and determine activity of several enzymes of
the purine pathway in patients with immunodeficiency of unknown etiology and
also determine the presence of abnormalities of nucleoside and nucleotide
concentrations in cells and body fluids of such patients.

## Key facts

- **NIH application ID:** 3124720
- **Project number:** 5R01AI010343-17
- **Recipient organization:** NEW YORK UNIVERSITY
- **SAM.gov UEI:** NX9PXMKW5KW8
- **Principal Investigator:** ROCHELLE HIRSCHHORN
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 1988
- **Award amount:** $252,928
- **Award type:** 5
- **Project period:** 1974-12-01 → 1989-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/3124720

## Citation

> US National Institutes of Health, RePORTER application 3124720, ACTIVATION OF IMMUNOCOMPETENT LYMPHOCYTES (5R01AI010343-17). Retrieved via MORVS 2026-10-25 from https://api.morvs.ai/grant/nih/3124720. Licensed CC0.

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