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  1. AA&MDSIF announces PNH grant awards

    ... aplastic anemia . PNH is caused by a somatic mutation in the PIG-A gene , which is responsible for a cellular ... will be used to examine the functional effects of the PIG-A mutation in CD34+ cells and T- cells shared by PNH, MDS and aplastic anemia ...

    Article last updated 12/14/2016 - 11:41am.

  2. Donor Lymphocyte Infusion (DLI) of T-cells Genetically Modified With iCasp9 Suicide Gene

    ... intermediate or high cytogenetic risk factors or flt3 mutation . Patients with relapsed disease. Patients with primary induction ...

    Clinical Trial last updated 06/06/2016 - 12:41pm.

  3. Detection of Recurrent Mutations by Pooled Targeted Next-Generation Sequencing in MDS Patients Prior to Treatment with Hypomethylating Agents or Stem Cell Transplantation

    ... (14%) genes.   In Cohort 2, 37% of patients had a mutation in a splicing factor gene ( SF3B1 , U2AF1 , SRSF2 , or ... of patients in Cohort 2 than Cohort 1 had a genetic mutation associated with a poor prognosis (especially TP53 ). Also, fewer ...

    Research Review last updated 05/02/2016 - 9:26am.

  4. Pathogenesis of MDS: An overview of molecular and non-molecular aspects of the disease

    ... that 78% of MDS patients carry at least one somatic mutation .  Knowledge of these mutations may help in diagnosing patients as ...

    Research Review last updated 05/02/2016 - 9:14am.

  5. Personalized Natural Killer (NK) Cell Therapy in Cord Blood Transplantation (CBT)

    ... abn(17p), MLL gene re-arrangement and t (6;9)47, flt3 mutation positive and/or evidence of minimal residual disease by flow ...

    Clinical Trial last updated 06/06/2016 - 10:28am.

  6. Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies

    ... or high-risk cytogenetics , flt3 mutation positive and/or evidence of minimal residual disease by flow ...

    Clinical Trial last updated 06/06/2016 - 12:58pm.

  7. Scientific Symposium Co-Chair Richard Stone, MD Discusses Progress and New Directions in Bone Marrow Failure Disease Research

    ... stem cell development, or a telomeropathy caused by mutation in genes encoding for members of the telomere complex. A ...

    Interview last updated 04/29/2014 - 12:01pm.

  8. Azacytidine Prior to in Vivo T-cell Depleted Allo Stem Cell Transplant for Patients With Myeloid Malignancies in CR

    ... abnormalities); or Acute myeloid leukemia with Flt-3 mutation in complete hematologic remission; or Acute myeloid leukemia, ...

    Clinical Trial last updated 05/02/2016 - 11:10am.

  9. Immune System Suppression and Bone Marrow Failure

    ... DNMT3A  and  ASXL1 and another had a RUNX1 mutation . Telomeres were very short in all of the patients with monosomy 7. ...

    Research Review last updated 05/02/2016 - 9:15am.

  10. Overlap Syndromes: When PNH Appears with Aplastic Anemia or MDS

    ... platelet count, especially if they have the JAK2 mutation . Does PNH appear concurrently with the other diseases or does ...

    Interview last updated 05/14/2015 - 1:53pm.