Gene Therapy Treats a Boy with Sickle Cell Disease

This very exciting paper has just been published in the NEJM  376;9 nejm.org March 2, 2017.

With the Lentivirus having already been used in experimental mouse models with DBA [1], this study in a child with sickle cell disease further highlights the importance of gene therapy research.

Pekka Jaako published an article in 2014 where he used the lentivirus to enforce expression of RPS19 in RPS19 deficient DBA cells which cured the anemia and bone marrow failure.”Our study provides a proof of principle for the development of clinical gene therapy to cure ribosomal protein 19-deficient Diamond-Blackfan anemia” [1].

screen-shot-2017-03-03-at-10-19-03-pm

It is a personal opinion only, but, I do believe that gene therapy is where the cure for DBA lies. Our summarized review on the paper will follow shortly, but in the interim, here is a link to a Medscape article well worth reading. Click Here

 

References

  1. Jaako P et al. Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia. Haematologica, 2014; 99(12).

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