Kshitiz Singh

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

J. Joung, S. Seneca, J. Vermeesch, 2018, Nucleic acids research.

Gene Expression Profile in the Sandhoff Mouse Brain with Progression of Age

Kshitiz Singh, J. Walia, Zhilin Chen, 2022, Genes.

In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

Rajan Jain, K. Musunuru, P. Zoltick, 2021, Nature Communications.

Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes

D. Furling, S. Dastidar, J. Tipanee, 2021, Molecular therapy : the journal of the American Society of Gene Therapy.

Combined targeting of EGFR / HER promotes anti-tumor efficacy in subsets of KRAS mutant lung cancer resistant to single EGFR blockade

O. De Wever, I. Umelo, Kshitiz Singh, 2015 .

In utero CRISPR-mediated therapeutic editing of metabolic genes

Li Li, Kiran Musunuru, Rajan Jain, 2018, Nature Medicine.

In utero gene editing for monogenic lung disease

Kiran Musunuru, E. Morrisey, K. Musunuru, 2019, Science Translational Medicine.

In utero CRISPR-mediated therapeutic editing of metabolic genes

E. Morrisey, Rajan Jain, K. Musunuru, 2018, Nature Medicine.

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

S. Sarcar, M. Y. Rincon, H. Evens, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

HEMOPHILIA GENE THERAPY: THE END OF THE BEGINNING?

T. VandenDriessche, M. Chuah, Kshitiz Singh, 2023, Human gene therapy.