Abstract

Chediak-Higashi syndrome (CHS) and Hermansky-Pudlak syndrome (HPS) are fatal human genetic disorders characterized by structural and functional defects of multiple cytoplasmic organelles -- melanosomes, lysosomes, and granules. We have cloned the genes for both CHS and HPS and are currently preparing antisera to the corresponding proteins. We wish to define the ina-acellular location of the CHS and BPS proteins (which are entirely novel), in both normal and mutant cells, and to determine whether these proteins interact with each other and with other cellular components. We will immunostain fissue samples using our antisera and determine cellular localization of CHS and HPS using the confocal microscope at the MM. These studies should help elucidate the pathogenesis of these disorders and thus guide eventual design of rational therapies. SCIENTIFXC SUBPROJECT GRANT NUMBER: P41RR00570-27

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
Biotechnology Resource Grants (P41)
Project #
3P41RR000570-28
Application #
6278495
Study Section
Project Start
1998-07-01
Project End
2000-06-30
Budget Start
1997-10-01
Budget End
1998-09-30
Support Year
28
Fiscal Year
1998
Total Cost
Indirect Cost

  Institution

Name
University of Wisconsin Madison
Department
Type
DUNS #
161202122
City
Madison
State
WI
Country
United States
Zip Code
53715

  Publications

Malecki, Marek; Putzer, Emily; Sabo, Chelsea et al. (2014) Directed cardiomyogenesis of autologous human induced pluripotent stem cells recruited to infarcted myocardium with bioengineered antibodies. Mol Cell Ther 2:
Malecki, Marek (2014) 'Above all, do no harm': safeguarding pluripotent stem cell therapy against iatrogenic tumorigenesis. Stem Cell Res Ther 5:73
Mavroudi, Maria; Zarogoulidis, Paul; Porpodis, Konstantinos et al. (2014) Stem cells' guided gene therapy of cancer: New frontier in personalized and targeted therapy. J Cancer Res Ther (Manch) 2:22-33
Malecki, Marek; LaVanne, Christine; Alhambra, Dominique et al. (2013) Safeguarding Stem Cell-Based Regenerative Therapy against Iatrogenic Cancerogenesis: Transgenic Expression of DNASE1, DNASE1L3, DNASE2, DFFB Controlled By POLA1 Promoter in Proliferating and Directed Differentiation Resisting Human Autologous Pluripotent J Stem Cell Res Ther Suppl 9:
Malecki, Marek; Tombokan, Xenia; Anderson, Mark et al. (2013) TRA-1-60(+), SSEA-4(+), POU5F1(+), SOX2(+), NANOG(+) Clones of Pluripotent Stem Cells in the Embryonal Carcinomas of the Testes. J Stem Cell Res Ther 3:
Malecki, Marek (2013) Improved targeting and enhanced retention of the human, autologous, fibroblast-derived, induced, pluripotent stem cells to the sarcomeres of the infarcted myocardium with the aid of the bioengineered, heterospecific, tetravalent antibodies. J Stem Cell Res Ther 3:
Malecki, Marek; Dahlke, Jessica; Haig, Melissa et al. (2013) Eradication of Human Ovarian Cancer Cells by Transgenic Expression of Recombinant DNASE1, DNASE1L3, DNASE2, and DFFB Controlled by EGFR Promoter: Novel Strategy for Targeted Therapy of Cancer. J Genet Syndr Gene Ther 4:152
Zarogoulidis, Paul; Darwiche, Kaid; Sakkas, Antonios et al. (2013) Suicide Gene Therapy for Cancer - Current Strategies. J Genet Syndr Gene Ther 4:
Malecki, Marek; Sabo, Chelsea; Putzer, Emily et al. (2013) Recruitment and retention of human autologous CD34+ CD117+ CD133+ bone marrow stem cells to infarcted myocardium followed by directed vasculogenesis: Novel strategy for cardiac regeneration. Mol Cell Ther 1:
Malecki, Marek; Malecki, Bianca (2012) Routing of Biomolecules and Transgenes' Vectors in Nuclei of Oocytes. J Fertili In Vitro 2012:108-118

Showing the most recent 10 out of 24 publications