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CNS Therapy Development for Lysosomal Storage Disorders (R01)
Release Date: July 2, 2004
Announcement Number: PAS-04-120
Application Receipt Date:
October 1, 2004
February 1, 2005
June 1, 2005
October 1, 2005
February 1, 2006
June 1, 2006
October 1, 2006
Funding Contact: Danilo Tagle, Ph.D.
Program Area: Neurogenetics
Brief Description:
The goal of this Program Announcement 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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