Pancreatic Islet and PARAthyroid Co-Transplantation for Treatment of Diabetes in IntraMuscular Site: PARADIGM

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Transplantation of beta cells, contained in donor pancreatic islets, can reverse the symptoms of diabetes.  However, due to a poor islet survival rate, transplants require islets from multiple donors.  Since…

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Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

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Cystinosis is a rare disease that primarily affects children and young adults, and leads to premature death, usually in early adulthood.  Patients inherit defective copies of a gene called CTNS,…

Continue ReadingPhase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

Induction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients

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Dr. Meyer, and his team at Stanford University, will use a combination of healthy donor stem cells and the patient's own regulatory T cells (Tregs), to train the patient's immune…

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A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

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Sangamo, Inc. is testing genetically engineered blood stem cells for the treatment of beta-thalassemia, a severe form of anemia caused by mutations in the hemoglobin gene. This genetic disorder requires…

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A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC

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The five-year survival rate for people diagnosed with the most advanced stage of non-small cell lung cancer (NSCLC) is between one and 10 percent. To address this devastating condition, UCLA researchers…

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Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells and Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

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UC San Francisco researchers aim to re­pair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can…

Continue ReadingGene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells and Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies

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Chimeric Antigen Receptor (CAR) T Cell Therapy is an innovative cancer therapy with very encouraging response rates in patients. The therapy works by isolating a patient's own T cells (a…

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Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients

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Patients who receive kidney transplants must take life-long immunosuppressive drugs to prevent their immune system from rejecting the transplant. Over time, these drugs are toxic and can increase a patient's…

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