Danielle N. Clark

Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

Michael L Kaufman, D. Kohn, Xiaoyan Wang, 2021, Molecular therapy. Methods & clinical development.

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.

Joseph D. Long, D. Kohn, Y. Kurmangaliyev, 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells

Y. Shaul, D. Kohn, R. Hollis, 2020, Frontiers in Genome Editing.

Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair

Michael L Kaufman, J. Corn, S. Fitz-Gibbon, 2018, Stem cells.

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

Michael L Kaufman, D. Kohn, Stephen Wandro, 2019, Molecular therapy. Methods & clinical development.

Prolonged STAT1 activation in neurons drives a pathological transcriptional response

A. Filiano, Kavya Raghunathan, Li Xu, 2023, Journal of Neuroimmunology.