| Category | Funding IC | Project Number | Sub Project # | Project Title | PI Name | Org Name | State / Country | Amount |
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
5R01NS034568-13
|
|
STRATEGIES TO ENHANCE GENE TRANSFER TO THE CNS
|
DAVIDSON, BEVERLY
|
UNIVERSITY OF IOWA
|
IA
|
$323,419
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
5R01NS057412-02
|
|
Suppression of the Idua-W402X mutation in an MPS I-H mouse
|
BEDWELL, DAVID
|
UNIVERSITY OF ALABAMA AT BIRMINGHAM
|
AL
|
$317,188
|
|
Mucopolysaccharidoses (MPS)
|
NHLBI
|
5R01HL049997-14
|
|
Cellular approaches to reduce BMT complications
|
BLAZAR, BRUCE
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$354,382
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
1R43HD057631-01
|
|
Iduronidase Replacement Therapy of the Brain in Hurler's Syndrome
|
ZHANG, YUFENG
|
ARMAGEN TECHNOLOGIES, INC.
|
CA
|
$100,000
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
5R01NS038690-07
|
|
AAV Gene Therapy for the CNS in MPS VII
|
WOLFE, JOHN
|
CHILDREN'S HOSPITAL OF PHILADELPHIA
|
PA
|
$446,473
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
5R44HD052303-03
|
|
Recombinant Enzyme Fusion Protein for Lysosomal Storage Disorders
|
ZHANG, YUN
|
ARMAGEN TECHNOLOGIES, INC.
|
CA
|
$456,597
|
|
Mucopolysaccharidoses (MPS)
|
NIMH
|
5R43MH079542-02
|
|
Live-Cells Assays for Lysosomal Enzyme Activity
|
NALEWAY, JOHN
|
MARKER GENE TECHNOLOGIES
|
OR
|
$199,822
|
|
Mucopolysaccharidoses (MPS)
|
NIDDK
|
5R01DK068216-04
|
|
COMPREHENSIVE GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS VII
|
ROY-CHOWDHURY, JAYANTA
|
YESHIVA UNIVERSITY
|
NY
|
$324,718
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
5R01NS053808-04
|
|
Aptamer-directed crossing of the blood barrier for enzyme therapy of LSDs
|
NEUFELD, ELIZABETH
|
UNIVERSITY OF CALIFORNIA LOS ANGELES
|
CA
|
$318,046
|
|
Mucopolysaccharidoses (MPS)
|
OD
|
5R01NS053808-04
|
|
Aptamer-directed crossing of the blood barrier for enzyme therapy of LSDs
|
NEUFELD, ELIZABETH
|
UNIVERSITY OF CALIFORNIA LOS ANGELES
|
CA
|
$145,635
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
5R01NS054242-03
|
|
Intrathecal enzyme therapy for mucopolysaccharidosis I
|
DICKSON, PATRICIA
|
LA BIOMED RES INST/ HARBOR UCLA MED CTR
|
CA
|
$339,787
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
5R01NS056243-02
|
|
Stem Cell Transplantation for Neurogenetic Disease
|
WOLFE, JOHN
|
CHILDREN'S HOSPITAL OF PHILADELPHIA
|
PA
|
$356,374
|
|
Mucopolysaccharidoses (MPS)
|
NIDDK
|
2R01DK054481-13A2
|
|
Gene Therapy of Mucopolysaccharidosis VII
|
HASKINS, MARK
|
UNIVERSITY OF PENNSYLVANIA
|
PA
|
$464,571
|
|
Mucopolysaccharidoses (MPS)
|
NIDDK
|
5R01DK025759-30
|
|
Animal Models of Mucopolysaccharidosis
|
HASKINS, MARK
|
UNIVERSITY OF PENNSYLVANIA
|
PA
|
$408,077
|
|
Mucopolysaccharidoses (MPS)
|
NIDDK
|
5R01DK067859-09
|
|
Multiplex Analysis of Inborn Errors of Metabolism
|
TURECEK, FRANTISEK
|
UNIVERSITY OF WASHINGTON
|
WA
|
$287,477
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
2R01NS022376-21A1
|
|
Molecular Studies of Sanfilippo Syndrome Type B
|
NEUFELD, ELIZABETH
|
UNIVERSITY OF CALIFORNIA LOS ANGELES
|
CA
|
$336,875
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
5P01HD032652-13
|
0003
|
Lentiviral gene therapy for MPS type I
|
WHITLEY, CHESTER
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$205,926
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
5P01HD032652-13
|
0006
|
Sleeping beauty transposon for gene therapy
|
HACKETT, PERRY
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$201,576
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
5P01HD032652-13
|
9002
|
Core--Research animal care and medicine
|
GILLETT, CYNTHIA
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$137,501
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
5P01HD032652-13
|
9005
|
Core--Viral vector
|
MCIVOR, R
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$61,654
|
|
Mucopolysaccharidoses (MPS)
|
NICHD
|
5P01HD032652-13
|
9006
|
Core--Neurological services
|
LOW, WALTER
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$91,599
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
2P40RR002512-24A1
|
|
Referral Center - Animal models of human genetic disease
|
HASKINS, MARK
|
UNIVERSITY OF PENNSYLVANIA
|
PA
|
$346,188
|
|
Mucopolysaccharidoses (MPS)
|
NINDS
|
1R01NS064330-01
|
|
Genetic Therapy for CNS Manifestations in MPS I via BBB-targeted Protein Delivery
|
PAN, DAO
|
CHILDREN'S HOSPITAL MED CTR (CINCINNATI)
|
OH
|
$328,125
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
3P40RR002512-23S1
|
|
REFERRAL CENTER--ANIMAL MODELS OF HUMAN GENETIC DISEASE
|
HASKINS, MARK
|
UNIVERSITY OF PENNSYLVANIA
|
PA
|
$139,055
|
|
Mucopolysaccharidoses (MPS)
|
OD
|
2R56DK066448-06A1
|
|
RETROVIRAL VECTOR-MEDIATED LIVER GENE THERAPY FOR MPS I
|
PONDER, KATHERINE
|
WASHINGTON UNIVERSITY
|
MO
|
$240,436
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
5P51RR013986-10
|
6333
|
ADENO ASSOCIATED VIRUS (AAV) GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE II
|
BRUNETTI-PIERRI, NICOLA
|
SOUTHWEST FOUNDATION FOR BIOMEDICAL RES
|
TX
|
$127
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
2M01RR000046-48
|
6875
|
CLINICAL TRIAL: MAINTENANCE STUDY OF IDURONATE-2-SULFATASE REPLACEMENT THERAPY I
|
MUENZER, JOSEPH
|
UNIVERSITY OF NORTH CAROLINA CHAPEL HILL
|
NC
|
$153
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
2M01RR000046-48
|
6920
|
TKT024EXT: MPS II PATIENTS RECEIVING IDURONATE-2-SULFATASE ENZYME REPLACEMENT
|
MUENZER, JOSEPH
|
UNIVERSITY OF NORTH CAROLINA CHAPEL HILL
|
NC
|
$1,301
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
5M01RR000425-39
|
7393
|
CLINICAL TRIAL: A STUDY OF INTRATHECAL ENZYME REPLACEMENT THERAPY FOR SPINAL COR
|
DICKSON, PATRICIA
|
LA BIOMED RES INST/ HARBOR UCLA MED CTR
|
CA
|
$23,902
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
5M01RR000400-40
|
7469
|
CLINICAL TRIAL: PHASE II STUDY OF COMBINED LARONIDASE (ALDURAZYME) ENZYME REPLAC
|
ORCHARD, PAUL
|
UNIVERSITY OF MINNESOTA TWIN CITIES
|
MN
|
$8,392
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
5M01RR000188-44
|
7856
|
CLINICAL TRIAL: AN OPEN-LABEL EXTENSION OF STUDY TKT024 EVALUATING LONG-TERM SAF
|
ENG, CHRISTINE
|
BAYLOR COLLEGE OF MEDICINE
|
TX
|
$2,900
|
|
Mucopolysaccharidoses (MPS)
|
NCRR
|
5M01RR000064-44
|
8620
|
CLINICAL TRIAL: MPS II PATIENTS RECEIVING ENZYME REPLACEMENT THERAPY
|
VISKOCHIL, DAVID
|
UNIVERSITY OF UTAH
|
UT
|
$688
|
