Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

Kiran Musunuru

Cardiovascular Institute, Perelman School of Medicine, Division of Cardiology, University of Pennsylvania

Sarah A. Grandinette

Perelman School of Medicine at the University of Pennsylvania, Philadelphia

Xiao Wang

Taylor R. Hudson

Innovative Genomics Institute, University of California, Berkeley, Berkeley

Kevin Briseno

Innovative Genomics Institute, University of California, Berkeley, Berkeley

Anne Marie Berry

Perelman School of Medicine at the University of Pennsylvania, Philadelphia

Julia L. Hacker

Perelman School of Medicine at the University of Pennsylvania, Philadelphia

Alvin Hsu

Broad Institute of MIT and Harvard, Harvard University, Howard Hughes Medical Institute, Cambridge, MA

Rachel A. Silverstein

Logan T. Hille

Massachusetts General Hospital–Harvard Medical School, Boston

Aysel N. Ogul

Innovative Genomics Institute, University of California, Berkeley, Berkeley

Nancy A. Robinson-Garvin

Children’s Hospital of Philadelphia, Philadelphia

Juliana C. Small

Children’s Hospital of Philadelphia, Philadelphia

Sarah McCague

Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia, Philadelphia

Samantha M. Burke

Children’s Hospital of Philadelphia, Philadelphia

Christina M. Wright

Children’s Hospital of Philadelphia, Philadelphia

Sarah Bick

Children’s Hospital of Philadelphia, Philadelphia

Venkata Indurthi

Aldevron, Fargo, ND

Shweta Sharma

Michael Jepperson

Aldevron, Fargo, ND

Christopher A. Vakulskas

Michael Collingwood

Integrated DNA Technologies, Coralville, IA

Katie Keogh

Integrated DNA Technologies, Coralville, IA

Ashley Jacobi

Integrated DNA Technologies, Coralville, IA

Morgan Sturgeon

Integrated DNA Technologies, Coralville, IA

Christian Brommel

Integrated DNA Technologies, Coralville, IA

Ellen Schmaljohn

Integrated DNA Technologies, Coralville, IA

Gavin Kurgan

Integrated DNA Technologies, Coralville, IA

Thomas Osborne

Integrated DNA Technologies, Coralville, IA

He Zhang

Kyle Kinney

Integrated DNA Technologies, Coralville, IA

Garrett Rettig

Integrated DNA Technologies, Coralville, IA

Christopher J. Barbosa

Acuitas Therapeutics, Vancouver, BC, Canada

Sean C. Semple

Acuitas Therapeutics

Ying K. Tam

Cathleen Lutz

Jackson Laboratory, Bar Harbor, ME

Lindsey A. George

Children’s Hospital of Philadelphia, Philadelphia

Benjamin P. Kleinstiver

David R. Liu

Kim Ng

Children’s Hospital of Philadelphia, Philadelphia

Sadik H. Kassim

Petros Giannikopoulos

Mohamad-Gabriel Alameh

Fyodor D. Urnov

Rebecca C. Ahrens-Nicklas

Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Philadelphia