Disease Focus: Hearing Loss


A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

Modulation of the Wnt pathway to restore inner ear function

Research Objective We aim to identify drug regimens that stimulate endogenous progenitors in the inner to regenerate to restore hearing or balance functions. Impact Treatment for irreversible hearing loss and balance disorders is limited, a drug regimen to reverse is highly impactful. Major Proposed Activities Production of R-spondin proteins Drug testing in neonatal cochlear cultures […]

A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs

Research Objective Development of a screen using inner ear sensory hair cell-like cells made by direct lineage reprogramming, for discovering drugs to ameliorate hearing loss during cancer chemotherapy. Impact Hearing loss, both adult and pediatric, due to life-saving cisplatin chemotherapies. Lack of human inner ear hair cells for drug discovery purposes and disease modeling. Major […]

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication Mucopolysaccharidosis I (MPSI) is a rare disease that affects predominantly children. Untreated, these patients typically die by the age of 10. Therapeutic Mechanism The therapeutic […]

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 […]

Generation of inner ear sensory cells from human ES cells toward a cure for deafness

Hearing loss is the leading birth defect in the United States with ~3 children in 1,000 born with partial to profound compromise of auditory function. Debilitating hearing loss is estimated to affect ~4% of people under 45 years of age, and 34% of those 65 years or over. A major cause of why acquired hearing […]

Hair Cells and Spiral Ganglion Neuron Differentiation from Human Embryonic Stem Cells

Hair cells (HCs) convert sound and balance signals into electrical impulses in the inner ear, including the cochlea and the vestibular endorgans, with remarkable precision and sensitivity. Our long-term goal is to stimulate HC regeneration in human inner ears and to enable the functional innervations of HCs by neurons. Hair cells are terminally-differentiated cells. Once […]

Spotlight on Deafness: Welcoming Remarks

Robert Klein, J.D. and Claire Pomeroy, M.D., M.B.A., gave the welcoming remarks for the “Spotlight on Deafness,” an educational event presented at the CIRM Governing Board meeting on March 12, 2008. Klein is Chair of the CIRM Governing Board and President of Klein Financial Corporation. Pomeroy is Vice Chancellor for Human Health Sciences and Dean […]

Spotlight on Deafness: Seminar by Karen Doyle, M.D.

Karen Doyle, M.D., Ph.D., spoke at the “Spotlight on Deafness,” an educational event presented at the CIRM Governing Board meeting on March 12, 2008. ¬†Dolye briefly summarized the current clinical options for treating hearing loss and discussed the promise of stem cell research in overcoming the limitations of hearing loss treatment. ¬†Doyle is a professor […]

Spotlight on Deafness: Seminar by Ebenezer Yamoah, Ph.D.

Ebenezer N. Yamoah, Ph.D. spoke at the “Spotlight on Deafness,” an educational event presented at the CIRM Governing Board meeting on March 12, 2008. Yamoah overviewed the biological mechanisms of hearing and discussed his stem cell research, which seeks to regenerate hair cells of the inner ear in order to restore hearing. Yamoah has a […]