Genetic Engineering and Biotechnology News

Embryonic stem cells
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Stem Cells at the Frontier: Breakthroughs, Barriers, and What’s Next

Credit: luismmolina / iStock / Getty Images Plus

Panelists:

Image of Robert Signer, PhD

Robert Signer, PhD

Associate Professor, Medicine
University of California San Diego

Panelist

Image of Robert Signer, PhD

Robert Signer, PhD

Robert Signer, PhD, is a stem cell biologist whose research on protein synthesis and homeostasis in hematopoietic stem cells has opened the door to unchartered areas of cellular investigation. He is currently the deputy director of the Stem Cell Discovery Center and an associate professor of medicine in the Division of Regenerative Medicine at the University of California San Diego. Signer earned his PhD in cellular and molecular pathology in the lab of Kenneth Dorshkind, PhD, at the University of California Los Angeles, and a Bachelor of Applied Science in engineering science (biomedical) from the University of Toronto. He conducted postdoctoral studies with Sean Morrison, PhD, at the University of Texas Southwestern Medical Center. Signer has won several prestigious awards including the Janet Rowley Award from the International Society for Experimental Hematology, Scholar Awards from the American Society of Hematology and the V Foundation for Cancer Research, and the Distinguished International Young Investigator Award in Stem Cell Research.
Image of Stephanie Cherqui, PhD

Stephanie Cherqui, PhD

Professor, Department of Pediatrics
University of California San Diego

Panelist

Image of Stephanie Cherqui, PhD

Stephanie Cherqui, PhD

Stephanie Cherqui, PhD, is professor in the department of pediatrics, division of genetics at the University of California San Diego, and the director of the UC San Diego Gene Therapy Initiative. She received her PhD in 2002 at Necker Hospital in Paris, France, on a project focused on the identification and characterization of the gene involved in cystinosis, an inherited metabolic disorder. She then specialized in stem cells and gene therapy during her post-doctoral internship at The Scripps Research Institute where she was appointed assistant professor in 2009. In 2012, Cherqui joined the department of pediatrics at the University of California San Diego. She has a distinguished record of translating fundamental discoveries into clinical applications. Among her most significant achievements is the development of the first-in-human autologous hematopoietic stem cell gene therapy for cystinosis. Cherqui has leveraged these discoveries to expand her therapeutic approach to multiple disorders such as Friedreich’s ataxia, mucopolysaccharidosis type IIIC, and Alzheimer’s disease. She is the chair of the Cystinosis Stem Cell and Gene Therapy Consortium, the chair of the Scientific Review Board of the Cystinosis Research Foundation, and a member of the ASCGT Gene and Cell Therapy of Genetic and Metabolic Diseases committee.

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Stem cells play a crucial role in growth, development, and healing. In drug development, they are studied for their potential to treat diseases such as Parkinson’s disease, diabetes, and certain blood disorders. Despite their promise, stem cell research also raises challenges, including large-scale production, technical limitations, and issues related to safety and ethical concerns.

This GEN Live show will focus on new areas of stem cell research, including aging. It will bring together a panel of leading experts to unpack the latest advances and ongoing challenges in stem cells. Panelists will explore how stem cells are transforming disease modeling, drug discovery, and personalized medicine with perspectives on clinical trial progress and hurdles such as manufacturing and scalability, regulatory developments, and safety considerations.

A live Q&A session followed the presentations, offering a chance to pose questions to our expert panelists.

Produced with support from:

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GEN LiveMultimediaAging (Developmental biology)ConferencesDiabetesDrug development (Pharmacology)Hematologic diseasesParkinson's diseaseResearch and developmentStem cell