Leading the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 and Sandhoff diseases
Donate
444 downloads
NTSAD Research Initiative Program RFP 2023.pdf
1687 downloads
Gray-Edwards NTSAD Lay Summary 0919.docx
1708 downloads
NTSAD d'Azzo_Lay summary_ 2019.pdf
1651 downloads
Lay progress report_Jiang 12 months.pdf
1855 downloads
Lehotay-Final-Report-0719.pdf
1802 downloads
Gritti-Final-report-0719.pdf
Modified on 06 September 2018 6506 downloads
for web site - funded research projects 2002-18 updated 7-19-18.pdf
62 downloads
BioStrategiesAnnounces GM1 PhII-SBIR (1).pdf
166 downloads
RR August 2017.docx
157 downloads
Heat-Shock-Protein.jpg
Modified on 08 December 2017 2882 downloads
NTSAD Funded Research 2002-2017.pdf
498 downloads
Active Research Grants as of May%2c 2017.pdf
778 downloads
2017-Research-Booklet (1).pdf
This booklet is a compilation of summaries of research work in the field of lysosomal storage and leukodystrophy diseases. It was created to accompany the NTSAD Research Session at the 2017 Annual Family Conference in Dallas, Texas on March 31, 2017.
719 downloads
Modified on 16 September 2016 2747 downloads
Sjoberg-summary.docx
Goals of the proposal:
Impact of research: Pharmacological chaperones represent a new class of treatment for patients with diseases that result from destabilization, unfolding, or misfolding of a protein. The current GM2 mouse model is a hexosaminidase knock-out (hex-/-), which does not produce any of the target protein and therefore cannot be used to evaluate various chaperones ability to bind and stabilize the protein. Creation of a knock-in mouse with a point mutation, which is equivalent to a known human late onset mutation, will allow analysis of in vivo dosing effects of specific chaperones that have already been developed. The mouse model will be made available for other late onset research.
This grant is made available by the Katie and Allie Buryk Research Fund.
Modified on 16 August 2016 2875 downloads
BIOSTRATEGIES 12 mo NTSAD Summary Report.pdf
One-Year Progress Report to National Tay-Sachs & Allied Diseases Association
BioStrategies LCLectin-assisted transnasal delivery of corrective enzyme for GM1 gangliosidosis
Investigators: David N. Radin, Carole L Cramer, Alessandra d'Azzo (SJCRH)
Executive Summary:Genetic deficiencies in the lysosomal enzyme β-galactosidase (β-gal) lead to the neurodegenerative disease GM1 gangliosidosis. Traditional enzyme replacement therapy (ERT) approaches are ineffective because they fail to deliver sufficient enzyme to the brain. Our goal in this research, is to determine if the plant RTB lectin (sugar-binding protein) can help to carry β-gal across nasal mucosal surfaces and increase access to the brain using the mouse model of GM1. Fusion proteins linking β-gal with the RTB lectin will be produced in a plant-based protein production system and the purified protein will be administered trans-nasally to β-gal-/- mice to determine if active enzyme reaches the cells of the brain.
Modified on 17 June 2014 3879 downloads
12-month-Progress-Report_Lee-Snyder_6-2012.pdf
A progress report on the Therapeutic Potential of Human-Induced Pluripotent Stem Cells (IPSCs) in the Sandhoff Disease Mouse Model of Lysosomal Storage Disorders
Modified on 06 June 2014 2634 downloads
RFP-guidelines_12-2013.pdf
The request for proposals from NTSAD,
NTSAD is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies in the areas of neurodegenerative disorders affecting the CNS, especially lysosomal storage disorders (such as Tay-Sachs, GM-1, Sandhoff) and pediatric leukodystrophies (such as Canavan).
Modified on 06 June 2014 2885 downloads
PROGRESS-REPORT_Taurine-Conjugated-GM2_Li.pdf
Six Months Progress Report: Studies of Taurine-conjugated GM2 in Tay-Sachs Disease (Jan 2012).
Modified on 06 June 2014 2701 downloads
PROGRESS-REPORT_Therapeutic-Potential-Pluripotent-Stem-Cells-Sandhoff_Lee-Snyder.pdf
A Progress Report on the, "Therapeutic Potential of Human-Induced Pluripotent Stem Cells (IPSCs) in the Sandhoff Disease Mouse Model of Lysosomal Storage Disorders"
Modified on 06 June 2014 2742 downloads
Progress-Report_Platt_18-month.pdf
Progress Report: NTSAD/CTSF Grant: Platt, Platt and Cerundolo - February 2012
Modified on 06 June 2014 2687 downloads
Progress-Report_Maegawa_12-month-Summary.pdf
The progress report on MLD 6-12 months into research.
Modified on 06 June 2014 3017 downloads
Progress-Report_Maegawa_FINAL_MLD-lay-summary.pdf
A final summary on the Lay language and MLD.
Modified on 06 June 2014 2585 downloads
Progress-Report_corrected_NTSAD-Grant-1yr.pdf
The corrected final progress report for Gao.
Modified on 06 June 2014 2600 downloads
Progress-Report_Eichler-12-month_Outcome-Measures_2-2012.pdf
A Progress Report on, the “Clinical Outcome Measures for Gene Therapy Trial in Infantile and Juvenile GM2”.
Modified on 06 June 2014 2623 downloads
Funded-Research-Projects-2002-2013_one-page.pdf
A list of funded research projects from 2002- 2013 (the first page).
Modified on 06 June 2014 3316 downloads
Funded-Research-Projects-4-2-13-excluding-LSDRC.pdf
A list of the funded research projects excluding LSDRC.
Username
Password
Remember Me