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Items: 1 to 20 of 25


Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.

Humbert O, Radtke S, Samuelson C, Carrillo RR, Perez AM, Reddy SS, Lux C, Pattabhi S, Schefter LE, Negre O, Lee CM, Bao G, Adair JE, Peterson CW, Rawlings DJ, Scharenberg AM, Kiem HP.

Sci Transl Med. 2019 Jul 31;11(503). pii: eaaw3768. doi: 10.1126/scitranslmed.aaw3768.


Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.

Park SH, Lee CM, Dever DP, Davis TH, Camarena J, Srifa W, Zhang Y, Paikari A, Chang AK, Porteus MH, Sheehan VA, Bao G.

Nucleic Acids Res. 2019 May 31. pii: gkz475. doi: 10.1093/nar/gkz475. [Epub ahead of print]


Spatial control of in vivo CRISPR-Cas9 genome editing via nanomagnets.

Zhu H, Zhang L, Tong S, Lee CM, Deshmukh H, Bao G.

Nat Biomed Eng. 2019 Feb;3(2):126-136. doi: 10.1038/s41551-018-0318-7. Epub 2018 Nov 12.


A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing.

Li A, Lee CM, Hurley AE, Jarrett KE, De Giorgi M, Lu W, Balderrama KS, Doerfler AM, Deshmukh H, Ray A, Bao G, Lagor WR.

Mol Ther Methods Clin Dev. 2018 Dec 6;12:111-122. doi: 10.1016/j.omtm.2018.11.009. eCollection 2019 Mar 15.


Oracles and Query Lower Bounds in Generalised Probabilistic Theories.

Barnum H, Lee CM, Selby JH.

Found Phys. 2018;48(8):954-981. doi: 10.1007/s10701-018-0198-4. Epub 2018 Jul 12.


A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.

Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA.

Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6.


A no-go theorem for theories that decohere to quantum mechanics.

Lee CM, Selby JH.

Proc Math Phys Eng Sci. 2018 Jun;474(2214):20170732. doi: 10.1098/rspa.2017.0732. Epub 2018 Jun 27.


Towards Device-Independent Information Processing on General Quantum Networks.

Lee CM, Hoban MJ.

Phys Rev Lett. 2018 Jan 12;120(2):020504. doi: 10.1103/PhysRevLett.120.020504.


Efficient CRISPR/Cas9-Mediated Genome Editing Using a Chimeric Single-Guide RNA Molecule.

Butt H, Eid A, Ali Z, Atia MAM, Mokhtar MM, Hassan N, Lee CM, Bao G, Mahfouz MM.

Front Plant Sci. 2017 Aug 24;8:1441. doi: 10.3389/fpls.2017.01441. eCollection 2017.


CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.

Wang HX, Li M, Lee CM, Chakraborty S, Kim HW, Bao G, Leong KW.

Chem Rev. 2017 Aug 9;117(15):9874-9906. doi: 10.1021/acs.chemrev.6b00799. Epub 2017 Jun 22. Review.


CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies.

Gomes-Silva D, Srinivasan M, Sharma S, Lee CM, Wagner DL, Davis TH, Rouce RH, Bao G, Brenner MK, Mamonkin M.

Blood. 2017 Jul 20;130(3):285-296. doi: 10.1182/blood-2017-01-761320. Epub 2017 May 24.


Examination of CRISPR/Cas9 design tools and the effect of target site accessibility on Cas9 activity.

Lee CM, Davis TH, Bao G.

Exp Physiol. 2018 Apr 1;103(4):456-460. doi: 10.1113/EP086043. Epub 2017 Apr 12. Review.


Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease.

Jarrett KE, Lee CM, Yeh YH, Hsu RH, Gupta R, Zhang M, Rodriguez PJ, Lee CS, Gillard BK, Bissig KD, Pownall HJ, Martin JF, Bao G, Lagor WR.

Sci Rep. 2017 Mar 16;7:44624. doi: 10.1038/srep44624.


Genome editing for inborn errors of metabolism: advancing towards the clinic.

Schneller JL, Lee CM, Bao G, Venditti CP.

BMC Med. 2017 Feb 27;15(1):43. doi: 10.1186/s12916-017-0798-4. Review.


Design and Validation of CRISPR/Cas9 Systems for Targeted Gene Modification in Induced Pluripotent Stem Cells.

Lee CM, Zhu H, Davis TH, Deshmukh H, Bao G.

Methods Mol Biol. 2017;1498:3-21.


Analysis of gene repair tracts from Cas9/gRNA double-stranded breaks in the human CFTR gene.

Hollywood JA, Lee CM, Scallan MF, Harrison PT.

Sci Rep. 2016 Aug 25;6:32230. doi: 10.1038/srep32230.


Bounds on the power of proofs and advice in general physical theories.

Lee CM, Hoban MJ.

Proc Math Phys Eng Sci. 2016 Jun;472(2190):20160076.


Treating hemoglobinopathies using gene-correction approaches: promises and challenges.

Cottle RN, Lee CM, Bao G.

Hum Genet. 2016 Sep;135(9):993-1010. doi: 10.1007/s00439-016-1696-0. Epub 2016 Jun 17. Review.


A Burden of Rare Variants Associated with Extremes of Gene Expression in Human Peripheral Blood.

Zhao J, Akinsanmi I, Arafat D, Cradick TJ, Lee CM, Banskota S, Marigorta UM, Bao G, Gibson G.

Am J Hum Genet. 2016 Feb 4;98(2):299-309. doi: 10.1016/j.ajhg.2015.12.023.


The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells.

Lee CM, Cradick TJ, Bao G.

Mol Ther. 2016 Mar;24(3):645-54. doi: 10.1038/mt.2016.8. Epub 2016 Jan 19.

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