| Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I N Gomez-Ospina, SG Scharenberg, N Mostrel, RO Bak, S Mantri, ... Nature communications 10 (1), 4045, 2019 | 106 | 2019 |
| Engineering monocyte/macrophage− specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing SG Scharenberg, E Poletto, KL Lucot, P Colella, A Sheikali, TJ Montine, ... Nature Communications 11 (1), 3327, 2020 | 41 | 2020 |
| CRISPR/Cas9 genome engineering in engraftable human brain-derived neural stem cells DP Dever, SG Scharenberg, J Camarena, EJ Kildebeck, JT Clark, ... Iscience 15, 524-535, 2019 | 32 | 2019 |
| An SPNS1-dependent lysosomal lipid transport pathway that enables cell survival under choline limitation SG Scharenberg, W Dong, A Ghoochani, K Nyame, R Levin-Konigsberg, ... Science Advances 9 (16), eadf8966, 2023 | 12 | 2023 |
| Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I. Nat. Commun. 10, 4045 N Gomez-Ospina, SG Scharenberg, N Mostrel, RO Bak, S Mantri, ... | 7 | 2019 |
| A lysosomal lipid transport pathway that enables cell survival under choline limitation SG Scharenberg, W Dong, K Nyame, R Levin-Konigsberg, AR Krishnan, ... Biorxiv, 2022.11. 27.517422, 2022 | 3 | 2022 |
| Monocyte lineage-specific glucocerebrosidase expression in human hematopoietic stem cells: a universal genome editing strategy for Gaucher disease N Gomez-Ospina, SG Scharenberg, KL Lucot, A Sheikali, MH Porteus Molecular Genetics and Metabolism 129 (2), S64-S65, 2020 | 1 | 2020 |
| An SPNS1-Dependent Lysosomal Lipid Transport Pathway That Enables Cell Survival Under Choline Limitation W Dong, S Scharenberg, A Ghoochani, K Nyame, R Levin-Konigsberg, ... 2023 AIChE Annual Meeting, 2023 | | 2023 |
| Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins N Gomez-Ospina, MH Porteus, SG Scharenberg, A Amorin US Patent App. 18/010,773, 2023 | | 2023 |
| Genome-edited Human Hematopoietic Stem Cells Correct Lysosomal Storage Disorders E Poletto, SG Scharenberg, GB Channabasavaiah, M Porteus, ... ANNALS OF NEUROLOGY 88, S213-S214, 2020 | | 2020 |
| Author Correction: Engineering monocyte/macrophage− specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing SG Scharenberg, E Poletto, KL Lucot, P Colella, A Sheikali, TJ Montine, ... Nature Communications 11, 2020 | | 2020 |
| Engineering monocyte lineage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing: A universal strategy for genetic correction in … SG Scharenberg, K Lucot, N Mostrel, A Sheikali, MH Porteus, ... HUMAN GENE THERAPY 30 (11), A186-A186, 2019 | | 2019 |
| Genome Edited Human Hematopoietic Stem Cells Correct Lysosomal Storage Disorders: Proof-of-Concept and Safety Studies for Mucopolysaccharidosis Type I and Gaucher Disease N Gomez-Ospina, SG Scharenberg, N Mostrel, N Raj, L Attardi, S Khan, ... MOLECULAR THERAPY 27 (4), 329-329, 2019 | | 2019 |
| Engineering the Hematopoietic System for Lysosomal Storage Disorders: Correction of Mucopolysaccharidosis Type I Using Genome-Edited, Human Hematopoietic Stem and Progenitor Cells N Gomez-Ospina, S Scharenberg, N Mostrel, S Mantri, C Nicolas, ... MOLECULAR THERAPY 26 (5), 310-311, 2018 | | 2018 |
| Engineering blood stem cells for autologous transplants for lysosomal diseases: Correction of mucopolysaccharidosis type I using genome-edited hematopoietic stem and progenitor … N Gomez-Ospina, SG Scharenberg, S Mantri, C Nicolas, ROBO Bak, ... Molecular Genetics and Metabolism 123 (2), S54-S55, 2018 | | 2018 |
