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.
Who can apply:
Anyone/General Public
City Or Township Governments
County Governments
Federally Recognized Indian Tribal Governments
Independent School Districts
Individual/Family
Minority Group
Native American Organization
Non-Government - General
Nonprofits Having A 501(C)(3) Status With The IRS, Other Than Institutions Of Higher Education
Nonprofits That Do Not Have A 501(C)(3) Status With The IRS, Other Than Institutions Of Higher Education
Other Private Institution/Organization
Private Institutions Of Higher Education
Private Nonprofit Institution/Organization (Includes Institutions Of Higher Education, Hospitals)
Profit Organization
Public And State Controlled Institutions Of Higher Education
Public Housing Authorities/Indian Housing Authorities
Small Business (Less Than 500 Employees
Special District Governments
State (Includes District Of Columbia; Includes Institutions Of Higher Education And Hospitals)
U.S. Territories And Possessions (Includes Institutions Of Higher Education, Hospitals)
Eligible functional categories:
Funding Sources:
Extramural Research Programs in the Neurosciences and Neurological Disorders
More Information:
http://grants.nih.gov/grants/guide/pa-files/PAS-04-120.html
If you have problems accessing the full announcement, please contact:
NIH OER Webmaster
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