Human Stem Cell Use: Adult or Tissue Stem Cell
Stem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection
The AIDS virus infects and destroys cells of the immune system such that the bodies of infected individuals cannot fight infections or some cancers. If untreated HIV infection leads to death. Current therapies to stop virus replication in the body are expensive and can have side effects. They also do not eliminate the virus from […]
Beta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells
Disorders affecting the blood, including Sickle Cell Disease (SCD), are the most common genetic disorders in the world. SCD causes significant suffering and early death, despite major improvements in medical management and advances in understanding the complex disease-related biology. A bone marrow transplant (BMT) can greatly benefit patients with SCD, by providing a life-long source […]
A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia by Transplantation of Autologous Hematopoietic Stem Cells Transduced with the Lentiviral Vector LentiGlobin® Encoding the Human β-A-T87Q-…
[REDACTED] plans to carry out a Phase 1/2 study to evaluate the safety and efficacy of [REDACTED] for the treatment of β-Thalassemia Major(BTM). [REDACTED] consists of autologous patient hematopoietic stem cells(HSC) that have been genetically modified ex vivo with a lentiviral vector that encodes a therapeutic form of the β-globin gene. [REDACTED] is administered through […]
Enhancing hair cell regeneration in mouse and human inner ear
Hearing loss (HL) is a permanent sensory disorder affecting over 278 million people worldwide. In the US and California, 20% of individuals suffer from this sensory deficit. Those affected range from newborns (2 per 1000), to children aged 3-17 (5 per 1000), to adults including the elderly (~33% in aged 65-74 and ~50% >85 years […]
Stem cell therapy for inflammatory bowel disease
One of the most promising approaches that physicians foresee for treating human disease is regenerative medicine. A major aim in this field is to restore function by repairing damaged organs. Inflammatory bowel disease (IBD) is a chronic disease characterized by intermittent episodes of intestinal inflammation and disruption of the intestinal epithelial barrier. It causes significant […]
White matter neuroregeneration after chemotherapy: stem cell therapy for “chemobrain”
Chemotherapy for cancer is often life saving, but it also causes a debilitating syndrome of impaired cognition characterized by deficits in attention, concentration, information processing speed, multitasking and memory. As a result, many cancer survivors find themselves unable to return to work or function in their lives as they had before their cancer therapy. These […]
Characterization of Human Skeletal Muscle Stem Cells for Clinical Application
Skeletal muscle makes up 40% of our bodies, dictates our form, is responsible for our ability to move, express ourselves, eat, breath and to look around. Restoration or preservation of the body’s normal form and function is the central goal of regenerative medicine and the central focus of my clinical specialty of plastic and reconstructive […]
Tunable hydrogels for therapeutic delivery of multipotent stem cells.
Rationale: Skin ulcers represent the largest economic burden of all skin diseases. Human adult stem cell therapies for the treatment of chronic wounds have shown considerable promise. However, a delivery system is needed before they can be used clinically. Hydrogels have emerged as a promising vehicle for stem cell delivery because their 3-D structure and […]
The generation and expansion of tissue-engineered small intestine from human stem/ progenitor cells: a preclinical study of functional translation
This proposal aims to complete the preclinical steps to develop tissue-engineered intestine (TESI) as a functional replacement of the small intestine to treat short bowel syndrome (SBS). Common birth conditions especially those associated with prematurity result in SBS wherein 50-75% of the small intestine is gone. SBS children cannot get adequate nutrition and supportive medical […]
Regulation of Epidermal Self-Renewal and Differentiation by Histone and DNA Demethylases.
Tissue specific stem and progenitor cells exist to replenish the tissue it resides during normal homeostasis or during regeneration from a wound. Disease and aging leads to a depletion of these stem and progenitor cells, which can impede the ability of the body to regenerate itself. Thus, an understanding of the mechanisms of how tissue […]