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Modulation of BCL11A for treatment of hemoglobinopathies
US9885041B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9885041-B2 |
| Application number | US-201614987219-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jan 4, 2016 |
| Priority date | Sep 15, 2008 |
| Publication date | Feb 6, 2018 |
| Grant date | Feb 6, 2018 |
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- Title
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Abstract
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The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
First claim
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We claim: 1. A composition comprising a CD34+ hematopoietic progenitor cell that comprises a vector encoding a nucleic acid that inhibits BCL11A expression. 2. The composition of claim 1 , wherein the nucleic acid is a BCL11A specific RNA interference agent. 3. The composition of claim 2 , wherein the RNA interference agent comprises one or more of the nucleotide sequences set forth in SEQ ID NO:1-6. 4. The composition of claim 1 , wherein the composition further comprises a pharmaceutically acceptable carrier or diluent. 5. The composition of claim 1 , wherein the CD 34+ hematopoietic progenitor cell is expanded ex vivo. 6. The composition of claim 1 , wherein the CD 34+ hematopoietic progenitor cell is formulated for injection or infusion. 7. The composition of claim 2 , wherein the CD 34+ hematopoietic progenitor cell is formulated for injection or infusion. 8. The composition of claim 3 , wherein the CD 34+ hematopoietic progenitor cell is formulated for injection or infusion. 9. The composition of claim 4 , wherein the CD34+ hematopoietic progenitor cell is formulated for injection or infusion. 10. The composition of claim 5 , wherein the CD34+ hematopoietic progenitor cell is formulated for injection or infusion. 11. A population of CD34+ hematopoietic progenitor cells, wherein the CD 34+ hematopoietic progenitor cells comprise a vector encoding a nucleic acid that inhibits BCL11A expression. 12. The population of CD34+ hematopoietic progenitor cells of claim 11 , wherein the nucleic acid is a BCL11A specific RNA interference agent. 13. The population of CD34+ hematopoietic progenitor cells of claim 12 , wherein the RNA interference agent comprises one or more of the nucleotide sequences set forth in SEQ ID NO:1-6. 14. The population of CD34+ hematopoietic progenitor cells of claim 13 , wherein the CD 34+ hematopoietic progenitor cells are expanded ex vivo.
Assignees
Inventors
Classifications
viral genome or elements thereof as genetic vector · CPC title
Antianaemics · CPC title
Transcription factors · CPC title
Drugs for disorders of the blood or the extracellular fluid · CPC title
Proteins not provided for elsewhere · CPC title
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Frequently asked questions
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- What does patent US9885041B2 cover?
- The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
- Who is the assignee on this patent?
- Childrens Medical Center, Harvard College, Childrens Medical Ct Corp
- What technology area does this patent fall under?
- Primary CPC classification C12N15/113. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Feb 06 2018 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).