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MOLECULAR BASIS OF HUMAN GAMMA-GLOBIN GENE ACTIVATION.

Award Number: R01HL073455
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: HEALTH SERVICES
PERIOD OF PERFORMANCE START DATE: 07/25/2003
PERIOD OF PERFORMANCE END DATE: 06/30/2007

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MOLECULAR BASIS OF HUMAN GAMMA-GLOBIN GENE ACTIVATION. - DESCRIPTION (provided by applicant): The long-term objective of the research is to develop reagents that activate human gamma globin gene transcription in the adult for treatment of beta-thalassemia and sickle cell anemia. It is known that expression of the fetal gamma globin genes greatly ameliorates the effects of these diseases, however it is not known how the human gamma globin genes are normally suppressed (or expressed at very low levels) when the expression of the human gamma globin genes switches to that of the delta and beta globin genes. It could be the result of an absence of transcription factors required for gamma globin gene expression or the presence of active suppressors of gamma globin expression or both. The aim of this application is therefore twofold: the characterization of the stage specific factors that are required for gamma globin gene expression and/or the stage specific factors that suppress gamma globin gene expression. Two independent in vivo approaches will be used. One uses chromatin precipitation to directly purify and sequence the proteins bound to the gamma globin gene promoters in the fetal stage and adult stage. The other is a functional assay to obtain single chain antibodies that switch on the gamma globin genes in the adult stage.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2006 ( Subtotal = $210,924 )
2006	2006	ERASMUS MC	POST OFFICE BOX 1738	ROTTERDAM		00000		NLD	Blood Diseases and Resources Research	000	4	8/22/2006	NON-COMPETING CONTINUATION	$210,924
														Subtotal = $210,924

Issue Date FY: 2005 ( Subtotal = $216,000 )
2005	2005	ERASMUS MC	POST OFFICE BOX 1738	ROTTERDAM		00000		NLD	Blood Diseases and Resources Research	000	3	9/9/2005	NON-COMPETING CONTINUATION	$216,000
														Subtotal = $216,000

Issue Date FY: 2004 ( Subtotal = $216,000 )
2004	2004	ERASMUS MC	POST OFFICE BOX 1738	ROTTERDAM		00000		NLD	Blood Diseases and Resources Research	000	2	7/13/2004	NON-COMPETING CONTINUATION	$216,000
														Subtotal = $216,000

Issue Date FY: 2003 ( Subtotal = $216,000 )
2003	2003	ERASMUS MC	POST OFFICE BOX 1738	ROTTERDAM		00000		NLD	Blood Diseases and Resources Research	000	1	7/25/2003	NEW	$216,000
														Subtotal = $216,000

Grand Total All Awards = $858,924

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MOLECULAR BASIS OF HUMAN GAMMA-GLOBIN GENE ACTIVATION.

Award Number: R01HL073455

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: HEALTH SERVICES

PERIOD OF PERFORMANCE START DATE: 07/25/2003

PERIOD OF PERFORMANCE END DATE: 06/30/2007

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