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Tisagenlecleucel in Children and Young Adults with B‐Cell Lymphoblastic Leukemia
TLDR
In this global study of CAR T‐cell therapy, a single infusion of tisagenlecleucel provided durable remission with long‐term persistence in pediatric and young adult patients with relapsed or refractory B‐cell ALL, with transient high‐grade toxic effects. Expand
NOD/SCID/gamma(c)(null) mouse: an excellent recipient mouse model for engraftment of human cells.
TLDR
It is suggested that multiple immunological dysfunctions, including cytokine production capability, in addition to functional incompetence of T, B, and NK cells, may lead to the high engraftment levels of xenograft in NOD/SCID/gamma(c)(null) mice. Expand
Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy
TLDR
Hematopoietic stem cell–specific microRNAs allow regulation of therapeutic transgene expression and enable effective gene therapy of globoid cell leukodystrophy and provide a basis for improvements in HSPC-mediated gene therapy and may offer patients a new therapeutic option that resembles a scratch more than a chop. Expand
NOD / SCID / cnull mouse : an excellent recipient mouse model for engraftment of human cells
c null mice and NOD/Shi-scid mice. When human CD34 cells from umbilical cord blood were transplanted into this strain, the engraftment rate in the peripheral circulation, spleen, and bone marrow wereExpand
Complete reconstitution of human lymphocytes from cord blood CD34+ cells using the NOD/SCID/gammacnull mice model.
TLDR
Results of ontogenic, functional, and histologic examination results of reconstituted human lymphocytes in NOD/SCID/ gammacnull mice after the transplantation of human cord blood (CB) CD34+ cells revealed that human lymphocyte formed organized structures in spleen and thymus that were analogous to those seen in humans. Expand
Attenuation of miR-126 Activity Expands HSC In Vivo without Exhaustion
TLDR
It is established that miR-126 sets a threshold for HSC activation and thus governs HSC pool size, demonstrating the importance of miRNA in the control of HSC function. Expand
Hyperinsulinism-hyperammonemia syndrome caused by mutant glutamate dehydrogenase accompanied by novel enzyme kinetics
TLDR
A novel variant GDH is identified in a patient with a more severe form of Hyperinsulinism-hyperammonemia syndrome and the mutation is located outside the GTP-binding site and the patient’s GDH shows consistently higher activity, even in the absence of allosteric effectors, which further support the hypothesis that the activating mutation ofGDH is the cause of HHS. Expand
Successful cord blood transplantation using a reduced‐intensity conditioning regimen for advanced childhood‐onset cerebral adrenoleukodystrophy
Awaya T, Kato T, Niwa A, Hiramatsu H, Umeda K, Watanabe K‐i, Shibata M, Yamanaka Y, Maruya E, Saji H, Nakahata T, Adachi S. Successful cord blood transplantation using a reduced‐intensityExpand
Clonal selection in xenografted TAM recapitulates the evolutionary process of myeloid leukemia in Down syndrome.
TLDR
The results suggest that genetically heterogeneous subclones with varying leukemia-initiating potential already exist in the neonatal TAM phase, and ML-DS may develop from a pool of such minor clones through clonal selection. Expand
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