Compositions and methods for the treatment of hemoglobinopathies

Compositions and methods for the treatment of hemoglobinopathies

  • CN 104,284,669 A
  • Filed: 02/22/2013
  • Published: 01/14/2015
  • Est. Priority Date: 02/24/2012
  • Status: Active Application
First Claim
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1. one kind is used for the treatment of the compositions of hemoglobinopathy, comprise one or more endonucleases be selected from homing endonuclease (HE) and CRISPR endonuclease, the nucleotide sequence in wherein often kind of described endonuclease is bonded to and is selected from Bcl11a coding region, Bcl11a Gene regulation district, adult'"'"'s betaglobulin locus, fetal hemoglobin (HbF) silent region, Bcl11a-regulate HbF silent region, gamma globulin gene promoter, δ

  • -Globulin gene promoter and betaglobulin gene mutation site.

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