Research Focus

Graduate Staff; NE Rotating Housestaff

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1. Repair of the sickle-cell disease mutation in human stem cells using CRISPR/Cas9 and single-stranded oligodeoxynucleotides

   Meeting Abstract

   Mansilla-Soto, J.; Leyfman, Y.; Sadelain, M.
   Molecular Therapy (2015)

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2. beta-Globin gene editing in human cells using TALENs and ssDNA oligonucleotides: Towards a gene repair approach for sickle cell anemia and beta-thalassemias

   Meeting Abstract

   Mansilla-Soto, J.; Socci, N.; Leyfman, Y.; Sadelain, M.
   Molecular Therapy (2014)

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