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Sarepta Therapeutics, Inc. Message Board

  • musherga musherga May 7, 2014 12:21 PM Flag

    missense therapy

    can SRPT morpholinos be used to correct missense mutations?

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    • I should be more specific. Point mutation e.g. sickle cell anemia.

      • 2 Replies to musherga
      • Mush,

        If interested in sickle cell, I suggest you look at SGMO and the single treatment curative program they recently partnered with Biogen-Idec to go after hemaglobinopathies such as Beta-Thalassemia and sickle cell. IMHO a SRPT exon skipping RX, even if possible for sickle cell, couldn't compete with SGMO's approach.
        December 9, 2013
        Sangamo BioSciences Announces First Presentation Of Data From ZFP Therapeutic Program For Treatment Of Sickle Cell Disease And Beta-Thalassemia At American Society Of Hematology Meeting
        Novel Genome-Editing Approach Offers a Potential Cure for Both Diseases
        RICHMOND, Calif., Dec. 9, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of preclinical data from its ZFP Therapeutic program for the treatment and potential cure of both sickle cell disease (SCD) and beta-thalassemia. The data demonstrate that Sangamo's approach, using its proprietary and highly specific zinc finger nuclease (ZFN) gene-editing technology, enables permanent increase in the expression of fetal gamma-globin in adult red blood cells (RBCs). This increase restores the normal balance of globin proteins that together form the oxygen-carrying hemoglobin of RBCs. In addition, the pre-clinical studies demonstrate that this can be accomplished at clinical-scale reproducibly achieving high levels (up to 80 percent) of gene editing in hematopoietic stem cells (HSCs).

        "These preclinical data demonstrate that Sangamo's precise ZFN genome editing technology enables a unique approach that can be used to treat, and potentially cure, both sickle cell disease and beta-thalassemia," said Mark Walters, M.D., Director of Blood and Marrow Transplantation at Children's Hospital & Research Center Oakland, and a leader of one of the clinical teams that will be conducting the first Phase 1 clinical trial of this ZFP Therapeutic in transfusion-dependent beta thalassemia patients. "The modification process is extremely efficient and scalable.

      • A single "base pair" mutation changes one amino acid in a polypeptide. One base can be skipped over to correct a diseased protein.

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