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Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling
Noggin/SB431542-based neural induction should facilitate the use of hES and hiPS cells in regenerative medicine and disease modeling and obviate the need for protocols based on stromal feeders or embryoid bodies. Expand
Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia
CD19 CAR T cell therapy induces complete remissions in 88% of 16 adult patients with relapsed or refractory acute lymphoblastic leukemia. CARving Out a Niche for CAR T Cell Immunotherapy Relapsed orExpand
CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia
The results demonstrate the marked antitumor efficacy of 19-28z CAR-modified T cells in patients with relapsed/refractory B-ALL and the reliability of this therapy to induce profound molecular remissions, forming a highly effective bridge to potentially curative therapy with subsequent allo-HSCT. Expand
Safety and persistence of adoptively transferred autologous CD19-targeted T cells in patients with relapsed or chemotherapy refractory B-cell leukemias.
It is concluded that this adoptive T-cell approach is promising and more likely to show clinical benefit in the setting of prior conditioning chemotherapy and low tumor burden or minimal residual disease. Expand
The basic principles of chimeric antigen receptor design.
This review focuses on the design ofCARs, including the requirements for optimal antigen recognition and different modalities to provide costimulatory support to targeted T cells, which include the use of second- and third generation CARs, costimulation ligands, chimericcostimulatory receptors, and cytokines. Expand
Long‐Term Follow‐up of CD19 CAR Therapy in Acute Lymphoblastic Leukemia
A phase 1 trial involving adults with relapsed B‐cell ALL who received an infusion of autologous T cells expressing the 19‐28z CAR at the Memorial Sloan Kettering Cancer Center found that patients with a low disease burden before treatment had markedly enhanced remission duration and survival and had a markedly lower incidence of the cytokine release syndrome and neurotoxic events after infusion. Expand
Conserved vertebrate mir-451 provides a platform for Dicer-independent, Ago2-mediated microRNA biogenesis
It is shown that maturation of miR-451, a functional miRNA that is perfectly conserved among vertebrates, is independent of Dicer, and the mir-451 backbone is amenable to reprogramming, permitting vector-driven expression of diverse functional miRNAs in the absence of Dacer. Expand
Involvement of granulocyte-macrophage colony-stimulating factor in pulmonary homeostasis.
It is demonstrated that GM- CSF is not an essential growth factor for basal hematopoiesis and an unexpected, critical role for GM-CSF in pulmonary homeostasis is revealed. Expand
Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection
It is demonstrated that directing a CD19-specific CAR to the T- cell receptor α constant (TRAC) locus not only results in uniform CAR expression in human peripheral blood T cells, but also enhances T-cell potency, with edited cells vastly outperforming conventionally generated CAR T cells in a mouse model of acute lymphoblastic leukaemia. Expand
Modeling Pathogenesis and Treatment of Familial Dysautonomia using Patient Specific iPSCs
The derivation of patient-specific FD-iPSCs and the directed differentiation into cells of all three germ layers including peripheral neurons are reported, illustrating the promise of iPSC technology for gaining new insights into human disease pathogenesis and treatment. Expand