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Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.

Bak RO, Dever DP, Reinisch A, Cruz Hernandez D, Majeti R, Porteus MH.

Elife. 2017 Sep 28;6. pii: e27873. doi: 10.7554/eLife.27873.


The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation.

Dever DP, Porteus MH.

Curr Opin Hematol. 2017 Nov;24(6):481-488. doi: 10.1097/MOH.0000000000000385. Review.


One-Step Biallelic and Scarless Correction of a β-Thalassemia Mutation in Patient-Specific iPSCs without Drug Selection.

Liu Y, Yang Y, Kang X, Lin B, Yu Q, Song B, Gao G, Chen Y, Sun X, Li X, Bu L, Fan Y.

Mol Ther Nucleic Acids. 2017 Mar 17;6:57-67. doi: 10.1016/j.omtn.2016.11.010. Epub 2016 Dec 10.


Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.

Ginn SL, Hallwirth CV, Liao SH, Teber ET, Arthur JW, Wu J, Lee HC, Tay SS, Hu M, Reddel RR, McCormack MP, Thrasher AJ, Cavazzana M, Alexander SI, Alexander IE.

Mol Ther Nucleic Acids. 2017 Mar 17;6:1-14. doi: 10.1016/j.omtn.2016.11.011. Epub 2016 Dec 10.


LMO2 and IL2RG synergize in thymocytes to mimic the evolution of SCID-X1 gene therapy-associated T-cell leukaemia.

Ruggero K, Al-Assar O, Chambers JS, Codrington R, Brend T, Rabbitts TH.

Leukemia. 2016 Sep;30(9):1959-62. doi: 10.1038/leu.2016.116. Epub 2016 May 12. No abstract available.


Use of genome-editing tools to treat sickle cell disease.

Tasan I, Jain S, Zhao H.

Hum Genet. 2016 Sep;135(9):1011-28. doi: 10.1007/s00439-016-1688-0. Epub 2016 Jun 1. Review.


A novel intranuclear RNA vector system for long-term stem cell modification.

Ikeda Y, Makino A, Matchett WE, Holditch SJ, Lu B, Dietz AB, Tomonaga K.

Gene Ther. 2016 Mar;23(3):256-62. doi: 10.1038/gt.2015.108. Epub 2015 Dec 3.


Adult stem cells and tissue engineering strategies for salivary gland regeneration: a review.

Yoo C, Vines JB, Alexander G, Murdock K, Hwang P, Jun HW.

Biomater Res. 2014 Jul 24;18:9. doi: 10.1186/2055-7124-18-9. eCollection 2014. Review.


Genetic barcode sequencing for screening altered population dynamics of hematopoietic stem cells transduced with lentivirus.

Zanatta DB, Tsujita M, Borelli P, Aguiar RB, Ferrari DG, Strauss BE.

Mol Ther Methods Clin Dev. 2014 Nov 19;1:14052. doi: 10.1038/mtm.2014.52. eCollection 2014.


Thymic expression of a T-cell receptor targeting a tumor-associated antigen coexpressed in the thymus induces T-ALL.

Cui Y, Onozawa M, Garber HR, Samsel L, Wang Z, McCoy JP, Burkett S, Wu X, Aplan PD, Mackall CL.

Blood. 2015 May 7;125(19):2958-67. doi: 10.1182/blood-2014-10-609271. Epub 2015 Mar 26.


Supramolecular nanosubstrate-mediated delivery for reprogramming and transdifferentiation of mammalian cells.

Hou S, Choi JS, Chen KJ, Zhang Y, Peng J, Garcia MA, Yu JH, Thakore-Shah K, Ro T, Chen JF, Peyda P, Fan G, Pyle AD, Wang H, Tseng HR.

Small. 2015 Jun 3;11(21):2499-504. doi: 10.1002/smll.201402602. Epub 2015 Jan 22.


Intracellular trafficking of cationic liposome-DNA complexes in living cells.

Coppola S, Estrada LC, Digman MA, Pozzi D, Cardarelli F, Gratton E, Caracciolo G.

Soft Matter. 2012 Aug 14;8(30):7919-7927.


Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

Xie F, Ye L, Chang JC, Beyer AI, Wang J, Muench MO, Kan YW.

Genome Res. 2014 Sep;24(9):1526-33. doi: 10.1101/gr.173427.114. Epub 2014 Aug 5.


Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection.

Ye L, Wang J, Beyer AI, Teque F, Cradick TJ, Qi Z, Chang JC, Bao G, Muench MO, Yu J, Levy JA, Kan YW.

Proc Natl Acad Sci U S A. 2014 Jul 1;111(26):9591-6. doi: 10.1073/pnas.1407473111. Epub 2014 Jun 9.


Targeted genome editing in human repopulating haematopoietic stem cells.

Genovese P, Schiroli G, Escobar G, Tomaso TD, Firrito C, Calabria A, Moi D, Mazzieri R, Bonini C, Holmes MC, Gregory PD, van der Burg M, Gentner B, Montini E, Lombardo A, Naldini L.

Nature. 2014 Jun 12;510(7504):235-240. doi: 10.1038/nature13420. Epub 2014 May 28.


Promoted Growth of Brain Tumor by the Transplantation of Neural Stem/Progenitor Cells Facilitated by CXCL12.

Yao NW, Chen CC, Yen CT, Chang C.

Transl Oncol. 2014 May 23. pii: S1936-5233(14)00042-4. doi: 10.1016/j.tranon.2014.04.007. [Epub ahead of print]


Production and purification of the multifunctional enzyme horseradish peroxidase.

Spadiut O, Herwig C.

Pharm Bioprocess. 2013 Aug 1;1(3):283-295.


Quantitative measurement of intracellular transport of nanocarriers by spatio-temporal image correlation spectroscopy.

Coppola S, Pozzi D, De Sanctis SC, Digman MA, Gratton E, Caracciolo G.

Methods Appl Fluoresc. 2013;1(1). doi: 10.1088/2050-6120/1/1/015005.


A cut above the rest: targeted genome editing technologies in human pluripotent stem cells.

Li M, Suzuki K, Kim NY, Liu GH, Izpisua Belmonte JC.

J Biol Chem. 2014 Feb 21;289(8):4594-9. doi: 10.1074/jbc.R113.488247. Epub 2013 Dec 20. Review.


Screening for severe combined immunodeficiency in neonates.

Kelly BT, Tam JS, Verbsky JW, Routes JM.

Clin Epidemiol. 2013 Sep 16;5:363-9. doi: 10.2147/CLEP.S48890. eCollection 2013. Review.

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