2019
Friday Fri
Pediatric Grand Rounds (CME): Using the Blood to Treat the Brain: Therapeutic Genome Editing for Lysosomal Storage Disorders
Natalia Gomez-Ospina, MD, PhD - Stanford School of Medicine
The Stanford Department of Pediatrics Presents The Harvey J. Cohen, MD, PhD Endowed Lectureship in Pediatrics
Dramatic improvements in sequencing technologies have increased our ability to diagnose genetic diseases, while effective interventions lag behind. Genome editing, a mechanism to rewrite DNA sequences, serves as the basis for a new generation of precise genetic therapies that can ameliorate or cure single-gene disorders.
This is the last session of Pediatric Grand Rounds before winter break. Pediatric Grand Rounds will resume Friday, January 10
Speaker
Natalia Gomez-Ospina, M.D., Ph.D.
Taube Pediatric Neurodegenerative Disease Scholar
Assistant Professor, Department of Pediatrics
Division of Medical Genetics, and Division of Stem Cell and Regenerative Medicine
Stanford School of Medicine
Session Description
Dramatic improvements in sequencing technologies have increased our ability to diagnose genetic diseases, while effective interventions lag behind. Genome editing, a mechanism to rewrite DNA sequences, serves as the basis for a new generation of precise genetic therapies that can ameliorate or cure single-gene disorders. Here, we review the current status of genome editing for genetic diseases with a focus on lysosomal storage disorders (LSDs). We examine in the greater detail, the rationale and current evidence supporting the clinical development of autologous transplantation of genome-edited hematopoietic stem cells to treat patients with LSDs, and the potential implications for using this as an adaptable platform for delivering therapeutic proteins to the brain.
Education Goals
- Basics of genome editing: platforms, repair mechanisms, and approaches to delivery
- Lysosomal dysfunction as a cause of the childhood and adult-onset neurodegeneration
- Update on clinical testing of in vivo genome editing for lysosomal storage diseases
- Ex vivo genome editing of human hematopoietic stem cells for lysosomal storage diseases: proof of concept studies in MPSI, Gaucher, and Krabbe disease.
Location
Lucile Packard Children's Hospital, West Building Auditorium
725 Welch RoadStanford, CA 94304
CME Credit
Accreditation
The Stanford University School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Credit Designation
The Stanford University School of Medicine designates this live activity for a maximum of 1.00 AMA PRA Category 1 Credit(s)TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Cultural and Linguistic Competency
California Assembly Bill 1195 requires continuing medical education activities with patient care components to include curriculum in the subjects of cultural and linguistic competency. The planners and speakers of this CME activity have been encouraged to address cultural issues relevant to their topic area. The Stanford University School of Medicine Multicultural Health Portal also contains many useful cultural and linguistic competency tools including culture guides, language access information and pertinent state and federal laws. You are encouraged to visit the portal: http://lane.stanford.edu/portals/cultural.html
Contact Stanford Center for Continuing Medical Education for CME credit transcript. Email Magna Patel, RSS Manager at magna@stanford.edu or stanfordcme@stanford.edu.
Planner and Faculty Disclosure to Learners
In accordance with the standards of the Accreditation Council for Continuing Medical Education (ACCME), all speakers, planners and/or persons who can influence the CME content must disclose to learners any relationships with commercial interests providing products or services that are relevant to the content of the presentation. The following individual(s) HAVE indicated the following relationships:
Planner
Bertil Glader, MD
Contracted Research: Agios
The following speakers, planning committee members and/or persons who can influence CME content have indicated they have NO relationships with commercial industry to disclose relevant to the content of this CME activity:
Course Director
Alan Schroeder, MD, Associate Chief for Research, Division of Pediatric Hospital Medicine
Planners
Mary Leonard, MD, MSCE, Chair Department of Pediatrics
Matthew Porteus, MD, PhD, Associate Professor, Division of Stem Cell Transplantation and Regenerative Medicine
Neville H. Golden, MD, Chief, Division of Adolescent Medicine
Lisa Chamberlain, MD, MPH, Associate Professor, General Pediatrics
Minnie Dasgupta, MD, Chief Resident, Pediatric Residency Program
Speaker:
Natalia Gomez-Ospina, MD, PhD