Abstract

This Phase I trial will assess the effects of expressing recombinant IDS in 2 adults with the """"""""mild"""""""" form of Hunter syndrome. Peripheral blood lymphocytes (PBL) will be harvested by aspheresis and then transduced ex vivo with retrovirus vector L2SN (FDA BB-IND #5370). Transduced PBL will be then infused on a monthly basis. Evaluation of patients will include serial measurement of IDS enzyme, assessment of urine glycosaminoglycans, measurement of liver and spleen volumes by CT scan, pulmonary function tests, echocardiography and EKG.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000400-30
Application #
6278259
Study Section
Project Start
1997-12-01
Project End
1998-11-30
Budget Start
1997-10-01
Budget End
1998-09-30
Support Year
30
Fiscal Year
1998
Total Cost
Indirect Cost

  Institution

Name
University of Minnesota Twin Cities
Department
Type
DUNS #
168559177
City
Minneapolis
State
MN
Country
United States
Zip Code
55455

  Publications

Harbin, Michelle M; Zavala, Hanan; Ryder, Justin R et al. (2018) Associations of sex, age and adiposity in endothelium-independent dilation in children. Physiol Meas 39:045002
Arikawa, Andrea Y; Kaufman, Beth C; Raatz, Susan K et al. (2018) Effects of a parallel-arm randomized controlled weight loss pilot study on biological and psychosocial parameters of overweight and obese breast cancer survivors. Pilot Feasibility Stud 4:17
Foster, Eric D; Bridges, Nancy D; Feurer, Irene D et al. (2018) Improved Health-Related Quality of Life in a Phase 3 Islet Transplantation Trial in Type 1 Diabetes Complicated by Severe Hypoglycemia. Diabetes Care 41:1001-1008
Ketterl, Tyler G; Chow, Eric J; Leisenring, Wendy M et al. (2018) Adipokines, Inflammation, and Adiposity in Hematopoietic Cell Transplantation Survivors. Biol Blood Marrow Transplant 24:622-626
Writing Committee for the Type 1 Diabetes TrialNet Oral Insulin Study Group; Krischer, Jeffrey P; Schatz, Desmond A et al. (2017) Effect of Oral Insulin on Prevention of Diabetes in Relatives of Patients With Type 1 Diabetes: A Randomized Clinical Trial. JAMA 318:1891-1902
Kotlyar, Michael; Thuras, Paul; Hatsukami, Dorothy K et al. (2017) Sex differences in physiological response to the combination of stress and smoking. Int J Psychophysiol 118:27-31
Cole, Abigail J; Kuchnia, Adam J; Beckman, Lauren M et al. (2017) Long-Term Body Composition Changes in Women Following Roux-en-Y Gastric Bypass Surgery. JPEN J Parenter Enteral Nutr 41:583-591
Di Bisceglie, A M; Lombardero, M; Teckman, J et al. (2017) Determination of hepatitis B phenotype using biochemical and serological markers. J Viral Hepat 24:320-329
Beckman, Lauren M; Boullata, Joseph I; Fisher, Paige L et al. (2017) Evaluation of Lean Body Weight Equation by Dual-Energy X-Ray Absorptiometry Measures. JPEN J Parenter Enteral Nutr 41:392-397
Marwaha, A K; Panagiotopoulos, C; Biggs, C M et al. (2017) Pre-diagnostic genotyping identifies T1D subjects with impaired Treg IL-2 signaling and an elevated proportion of FOXP3+IL-17+ cells. Genes Immun 18:15-21

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