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PAS-06-202, CNS Therapy Development for Lysosomal Storage Disorders (R21)
Release Date: March 7, 2006
Announcement Number: PAS-06-202
Application Receipt Date:
June 1, 2006
October 1, 2006
February 16, 2006
June 16, 2006
October 16, 2006
Funding Contact: Danilo Tagle, Ph.D.
Program Area: Neurogenetics
Brief Description:
The goal of this Funding Opportunity Announcement (FOA) is to solicit applications on lysosomal storage disorders (LSDs) focused
on improving CNS treatment outcomes, enhancing the effectiveness of delivery and targeting of cells, enzymes, drugs and genes
into the brain, and developing novel therapeutic modalities, such as implantable biocapsules and micro-electro-mechanical
systems (MEMS)-based devices. Lysosomal storage disorders constitute a group of recessive genetic diseases resulting from
cellular enzymatic deficiencies of acid hydrolases that normally catalyze the metabolism of glycoproteins, glycolipids and
other macromolecules, or from defects in transporter proteins leading to pathogenic accumulation of these substances in lysosomes.
Treatment modalities for LSDs are currently limited to bone marrow transplantation (BMT) and enzyme replacement therapy (ERT).
These approaches while providing significant promise for treatment of the visceral manifestations of LSDs, do little to address
CNS pathologies for this group of disorders. Thus this announcement specifically encourages the transition from basic studies
in LSDs to translational research for improved delivery of therapeutic cells, proteins, genes, and small molecules across
the blood-brain barrier.
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