COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMO

专利名称：COMPOSITIONS AND METHODS FOR THE

TREATMENT OF HEMOGLOBINOPATHIES

发明人：BENDER, Michael A.,GROUDINE, Mark

T.,STODDARD, Barry L.,TAKEUCHI, Ryo

申请号：EP17178186.7申请日：20130222公开号：EP3272356A1公开日：20180124

专利附图：

摘要：Provided are compositions and methods for the treatment of

hemoglobinopathies such as thalassemias and sickle cell disease. Compositions and

methods include one or more endonuclease(s) or endonuclease fusion protein(s),including one or more homing endonuclease(s) and/or homing endonuclease fusionprotein(s) and/or CRISPR endonuclease(s) and/or CRISPR endonuclease fusion protein(s):(a) to disrupt a Bcl1 1a coding region; (b) to disrupt a Bcl1 1a gene regulatory region; (c)to modify an adult human β-globin locus; (d) to disrupt a HbF silencing DNA regulatoryelement or pathway, such as a Bcl1 1a-regulated HbF silencing region; (e) to mutate oneor more γ-globin gene promoter(s) to achieve increased expression of a γ-globin gene; (f)to mutate one or more δ-globin gene promoter(s) to achieve increased expression of aδ-globin gene; and/or (g) to correct one or more β-globin gene mutation(s).

申请人：Fred Hutchinson Cancer Research Center

地址：1100 Fairview Avenue North Seattle, WA 98109 US

国籍：US

代理机构：Griffin, Philippa Jane

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