Disease Focus: Pediatrics
Differentiation of Human Embryonic Stem Cells into Urothelium
Augmentation or replacement of the bladder is often necessary for the treatment of adults with bladder cancer and children with spinal cord injury or spina bifida. Current surgical techniques utilize segments of intestine or stomach as a substitute for bladder wall. Use of intestinal segments is associated with many complications including infection, stones, salt imbalance, […]
Self-renewal and senescence in iPS cells derived from patients with a stem cell disease
The discovery of induced pluripotent stem (iPS) cell technology promises to revolutionize our understanding of human disease and to allow the development of new cellular therapies for regenerative medicine applications. The ability to reprogram a patient’s fibroblasts to iPS cells creates the opportunity to expand human cells with a specific genetic defect and to study […]
Stem Cell Gene Therapy for Sickle Cell Disease
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” under conditions of low oxygen, occurs with a frequency of 1/500 African-Americans, and is also common in Hispanic-Americans, who comprise up to 5% of SCD patients in California. The median survival based on 1991 […]
iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa
Genetic skin diseases constitute a diverse group of several hundred diseases that affect up to 2% of the population and include common disease such as psoriasis, atopic dermatitis, and wound healing. Patients with one genetic disease, dystrophic Epidermolysis bullosa (EB), lack a normal collagen VII (COL7A1) gene and suffer from debilitating blistering and scarring that […]
Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons
One of the main objectives of stem cell biology is to create physiologically relevant cell types that can be used to either facilitate the study of or directly treat human disease. Tremendous progress towards these goals has been made in the area of motor neuron disease and spinal cord injury through the findings that motor […]
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
This trial proposes to replace SCID patients’ dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to make space in the bone marrow for the healthy transplanted stem cells to engraft. The Stanford team will instead test a safe, non-toxic protein called […]
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects
In ADA-SCID, allogeneic hematopoietic (blood) stem cell transplants from non-matched sibling donors are a high risk procedure. Additionally, the efficacy of chronic enzyme replacement therapy is uncertain in the long-term. A team at UCLA is using a patient’s own blood stem cells to try and rebuild the damaged immune systems of patients with ADA-SCID. They will use what’s […]
A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
X-linked Chronic Granulomatous Disease (X-CGD) is a rare immune disorder that prevents white blood cells from killing foreign invaders. This results in severe, recurrent infections that can impact quality and length of a patient’s life. X-CGD is usually diagnosed before age 5, but without treatment, children die before age 10. A team at UCLA is using the patient’s own genetically […]
Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease
Sickle cell disease (SCD) is caused by a genetic mutation in the hemoglobin gene which causes red blood cells to “sickle” under conditions of low oxygen. SCD affects 1:500 African-Americans and is also common in Hispanic-Americans. The median survival for patients with SCD is 42 years for males and 48 years for females. A team at UCLA […]