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Items: 1 to 50 of 283

1.

Correction to: Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization.

Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS.

Stem Cell Res Ther. 2019 Feb 12;10(1):52. doi: 10.1186/s13287-019-1147-7.

2.

Chemistry, manufacturing and controls for gene modified hematopoietic stem cells.

Soni S, Kohn DB.

Cytotherapy. 2019 Feb 8. pii: S1465-3249(18)30699-6. doi: 10.1016/j.jcyt.2018.12.001. [Epub ahead of print]

PMID:
30745225
3.

Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017.

Amatuni GS, Currier RJ, Church JA, Bishop T, Grimbacher E, Nguyen AA, Agarwal-Hashmi R, Aznar CP, Butte MJ, Cowan MJ, Dorsey MJ, Dvorak CC, Kapoor N, Kohn DB, Markert ML, Moore TB, Naides SJ, Sciortino S, Feuchtbaum L, Koupaei RA, Puck JM.

Pediatrics. 2019 Feb;143(2). pii: e20182300. doi: 10.1542/peds.2018-2300.

PMID:
30683812
4.

Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome.

Masiuk KE, Laborada J, Roncarolo MG, Hollis RP, Kohn DB.

Cell Stem Cell. 2019 Feb 7;24(2):309-317.e7. doi: 10.1016/j.stem.2018.12.003. Epub 2019 Jan 10.

PMID:
30639036
5.

Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice.

Kwon HS, Logan AC, Chhabra A, Pang WW, Czechowicz A, Tate K, Le A, Poyser J, Hollis R, Kelly BV, Kohn DB, Weissman IL, Prohaska SS, Shizuru JA.

Blood. 2019 Jan 7. pii: blood-2018-06-853879. doi: 10.1182/blood-2018-06-853879. [Epub ahead of print] No abstract available.

PMID:
30617195
6.

Gene therapy for blood diseases.

Kohn DB.

Curr Opin Biotechnol. 2018 Dec 29;60:39-45. doi: 10.1016/j.copbio.2018.11.016. [Epub ahead of print] Review.

PMID:
30599357
7.

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.

Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014. eCollection 2018 Dec 14.

8.

IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System.

Puig-Saus C, Parisi G, Garcia-Diaz A, Krystofinski PE, Sandoval S, Zhang R, Champhekar AS, McCabe J, Cheung-Lau GC, Truong NA, Vega-Crespo A, Komenan MDS, Pang J, Macabali MH, Saco JD, Goodwin JL, Bolon B, Seet CS, Montel-Hagen A, Crooks GM, Hollis RP, Campo-Fernandez B, Bischof D, Cornetta K, Gschweng EH, Adelson C, Nguyen A, Yang L, Witte ON, Baltimore D, Comin-Anduix B, Kohn DB, Wang X, Cabrera P, Kaplan-Lefko PJ, Berent-Maoz B, Ribas A.

Clin Cancer Res. 2019 Feb 1;25(3):1000-1011. doi: 10.1158/1078-0432.CCR-18-0963. Epub 2018 Nov 8.

PMID:
30409823
9.

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

Lomova A, Clark DN, Campo-Fernandez B, Flores-Bjurström C, Kaufman ML, Fitz-Gibbon S, Wang X, Miyahira EY, Brown D, DeWitt MA, Corn JE, Hollis RP, Romero Z, Kohn DB.

Stem Cells. 2019 Feb;37(2):284-294. doi: 10.1002/stem.2935. Epub 2018 Nov 27.

PMID:
30372555
10.

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.

Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD.

Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z.

11.

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.

Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman ML, Brown D, Mavilio F, Kohn DB.

Hum Gene Ther. 2018 Oct;29(10):1153-1166. doi: 10.1089/hum.2018.061.

PMID:
30198339
12.

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.

Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD, Grunebaum E.

J Allergy Clin Immunol. 2019 Mar;143(3):852-863. doi: 10.1016/j.jaci.2018.08.024. Epub 2018 Sep 5.

PMID:
30194989
13.

The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018).

Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Logan B, Griffith LM, Kohn DB, Pai SY, Notarangelo L, Shearer W, Prockop S, Kapoor N, Heimall J, Chaudhury S, Shyr D, Chandra S, Cuvelier G, Moore T, Shenoy S, Goldman F, Smith AR, Sunkersett G, Vander Lugt M, Caywood E, Quigg T, Torgerson T, Chandrakasan S, Craddock J, Dávila Saldaña BJ, Gillio A, Shereck E, Aquino V, DeSantes K, Knutsen A, Thakar M, Yu L, Puck JM.

J Allergy Clin Immunol. 2019 Jan;143(1):405-407. doi: 10.1016/j.jaci.2018.08.027. Epub 2018 Sep 5. No abstract available.

PMID:
30193840
14.

Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.

Hu P, Bi Y, Ma H, Suwanmanee T, Zeithaml B, Fry NJ, Kohn DB, Kafri T.

Gene Ther. 2018 Oct;25(7):454-472. doi: 10.1038/s41434-018-0039-2. Epub 2018 Sep 6.

PMID:
30190607
15.

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.

Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD.

Blood. 2018 Oct 25;132(17):1737-1749. doi: 10.1182/blood-2018-03-840702. Epub 2018 Aug 28.

PMID:
30154114
16.

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome.

Kuo CY, Long JD, Campo-Fernandez B, de Oliveira S, Cooper AR, Romero Z, Hoban MD, Joglekar AV, Lill GR, Kaufman ML, Fitz-Gibbon S, Wang X, Hollis RP, Kohn DB.

Cell Rep. 2018 May 29;23(9):2606-2616. doi: 10.1016/j.celrep.2018.04.103.

17.

B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation.

Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY.

Blood. 2018 Jun 28;131(26):2967-2977. doi: 10.1182/blood-2017-10-809822. Epub 2018 May 4.

PMID:
29728406
18.

Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.

Almarza Novoa E, Kasbekar S, Thrasher AJ, Kohn DB, Sevilla J, Nguyen T, Schwartz JD, Bueren JA.

J Allergy Clin Immunol Pract. 2018 Jul - Aug;6(4):1418-1420.e10. doi: 10.1016/j.jaip.2017.12.008. Epub 2018 Jan 20. No abstract available.

19.

Gene therapy comes of age.

Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M.

Science. 2018 Jan 12;359(6372). pii: eaan4672. doi: 10.1126/science.aan4672. Review.

PMID:
29326244
20.

Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells.

Long J, Hoban MD, Cooper AR, Kaufman ML, Kuo CY, Campo-Fernandez B, Lumaquin D, Hollis RP, Wang X, Kohn DB, Romero Z.

Mol Ther. 2018 Feb 7;26(2):468-479. doi: 10.1016/j.ymthe.2017.11.001. Epub 2017 Nov 9.

21.

Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.

Morgan RA, Gray D, Lomova A, Kohn DB.

Cell Stem Cell. 2017 Nov 2;21(5):574-590. doi: 10.1016/j.stem.2017.10.010. Review.

22.

Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.

Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC.

Blood. 2017 Dec 21;130(25):2718-2727. doi: 10.1182/blood-2017-05-781849. Epub 2017 Oct 11.

23.

Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization.

Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS.

Stem Cell Res Ther. 2017 Oct 2;8(1):217. doi: 10.1186/s13287-017-0652-9. Erratum in: Stem Cell Res Ther. 2019 Feb 12;10(1):52.

24.

Gene therapy: WAS (not) just for kids.

Kohn DB.

Blood. 2017 Sep 14;130(11):1278-1279. doi: 10.1182/blood-2017-08-798496. No abstract available.

25.

Gene Therapy.

Bauer DE, Kohn DB.

Hematol Oncol Clin North Am. 2017 Oct;31(5):xiii-xiv. doi: 10.1016/j.hoc.2017.07.001. Epub 2017 Aug 7. No abstract available.

PMID:
28895857
26.

Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy.

Kohn DB.

Hematol Oncol Clin North Am. 2017 Oct;31(5):721-735. doi: 10.1016/j.hoc.2017.06.006. Review.

PMID:
28895843
27.

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.

Bradford KL, Moretti FA, Carbonaro-Sarracino DA, Gaspar HB, Kohn DB.

J Clin Immunol. 2017 Oct;37(7):626-637. doi: 10.1007/s10875-017-0433-3. Epub 2017 Aug 25. Review.

PMID:
28842866
28.

Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.

Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, Kohn DB.

Cytotherapy. 2017 Sep;19(9):1096-1112. doi: 10.1016/j.jcyt.2017.06.002. Epub 2017 Jul 18.

PMID:
28733131
29.

Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells.

Masiuk KE, Brown D, Laborada J, Hollis RP, Urbinati F, Kohn DB.

Mol Ther. 2017 Sep 6;25(9):2163-2175. doi: 10.1016/j.ymthe.2017.05.023. Epub 2017 Jun 27.

30.

Generation of mature T cells from human hematopoietic stem and progenitor cells in artificial thymic organoids.

Seet CS, He C, Bethune MT, Li S, Chick B, Gschweng EH, Zhu Y, Kim K, Kohn DB, Baltimore D, Crooks GM, Montel-Hagen A.

Nat Methods. 2017 May;14(5):521-530. doi: 10.1038/nmeth.4237. Epub 2017 Apr 3.

31.

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.

Cooper AR, Lill GR, Shaw K, Carbonaro-Sarracino DA, Davila A, Sokolic R, Candotti F, Pellegrini M, Kohn DB.

Blood. 2017 May 11;129(19):2624-2635. doi: 10.1182/blood-2016-12-756734. Epub 2017 Mar 28.

32.

Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.

Shaw KL, Garabedian E, Mishra S, Barman P, Davila A, Carbonaro D, Shupien S, Silvin C, Geiger S, Nowicki B, Smogorzewska EM, Brown B, Wang X, de Oliveira S, Choi Y, Ikeda A, Terrazas D, Fu PY, Yu A, Fernandez BC, Cooper AR, Engel B, Podsakoff G, Balamurugan A, Anderson S, Muul L, Jagadeesh GJ, Kapoor N, Tse J, Moore TB, Purdy K, Rishi R, Mohan K, Skoda-Smith S, Buchbinder D, Abraham RS, Scharenberg A, Yang OO, Cornetta K, Gjertson D, Hershfield M, Sokolic R, Candotti F, Kohn DB.

J Clin Invest. 2017 May 1;127(5):1689-1699. doi: 10.1172/JCI90367. Epub 2017 Mar 27.

33.

New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing.

Kohn DB, Kuo CY.

J Allergy Clin Immunol. 2017 Mar;139(3):726-732. doi: 10.1016/j.jaci.2017.01.007. Review.

34.

How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).

Kohn DB, Gaspar HB.

J Clin Immunol. 2017 May;37(4):351-356. doi: 10.1007/s10875-017-0373-y. Epub 2017 Feb 14. Review.

PMID:
28194615
35.

Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases.

Bjurström CF, Mojadidi M, Phillips J, Kuo C, Lai S, Lill GR, Cooper A, Kaufman M, Urbinati F, Wang X, Hollis RP, Kohn DB.

Mol Ther Nucleic Acids. 2016;5:e351. doi: 10.1038/mtna.2016.52.

36.

Domain-swapped T cell receptors improve the safety of TCR gene therapy.

Bethune MT, Gee MH, Bunse M, Lee MS, Gschweng EH, Pagadala MS, Zhou J, Cheng D, Heath JR, Kohn DB, Kuhns MS, Uckert W, Baltimore D.

Elife. 2016 Nov 8;5. pii: e19095. doi: 10.7554/eLife.19095.

37.

Propagating Humanized BLT Mice for the Study of Human Immunology and Immunotherapy.

Smith DJ, Lin LJ, Moon H, Pham AT, Wang X, Liu S, Ji S, Rezek V, Shimizu S, Ruiz M, Lam J, Janzen DM, Memarzadeh S, Kohn DB, Zack JA, Kitchen SG, An DS, Yang L.

Stem Cells Dev. 2016 Dec 15;25(24):1863-1873. Epub 2016 Oct 18.

38.

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.

DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo SJ, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DI, Corn JE.

Sci Transl Med. 2016 Oct 12;8(360):360ra134.

39.

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.

Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ.

Hum Gene Ther. 2017 Jan;28(1):112-124. doi: 10.1089/hum.2016.064. Epub 2016 Sep 7.

40.

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.

Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz-Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, Hollis RP, Kohn DB.

Mol Ther. 2016 Sep;24(9):1561-9. doi: 10.1038/mt.2016.148. Epub 2016 Jul 29.

41.

Analyzing CRISPR genome-editing experiments with CRISPResso.

Pinello L, Canver MC, Hoban MD, Orkin SH, Kohn DB, Bauer DE, Yuan GC.

Nat Biotechnol. 2016 Jul 12;34(7):695-7. doi: 10.1038/nbt.3583. No abstract available.

42.

Primary Immune Deficiency Treatment Consortium (PIDTC) update.

Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E; workshop participants.

J Allergy Clin Immunol. 2016 Aug;138(2):375-85. doi: 10.1016/j.jaci.2016.01.051. Epub 2016 Apr 22. Review.

43.

Gene Therapy for the Treatment of Primary Immune Deficiencies.

Kuo CY, Kohn DB.

Curr Allergy Asthma Rep. 2016 May;16(5):39. doi: 10.1007/s11882-016-0615-8. Review.

44.

Ethical and regulatory aspects of genome editing.

Kohn DB, Porteus MH, Scharenberg AM.

Blood. 2016 May 26;127(21):2553-60. doi: 10.1182/blood-2016-01-678136. Epub 2016 Apr 6. Review.

45.

Genetic Tagging During Human Mesoderm Differentiation Reveals Tripotent Lateral Plate Mesodermal Progenitors.

Chin CJ, Cooper AR, Lill GR, Evseenko D, Zhu Y, He CB, Casero D, Pellegrini M, Kohn DB, Crooks GM.

Stem Cells. 2016 May;34(5):1239-50. doi: 10.1002/stem.2351. Epub 2016 Mar 28.

46.

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.

Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD.

Cell Stem Cell. 2016 Apr 7;18(4):533-40. doi: 10.1016/j.stem.2016.01.021. Epub 2016 Feb 11.

47.

Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells.

Hoban MD, Romero Z, Cost GJ, Mendel M, Holmes M, Kohn DB.

Curr Protoc Stem Cell Biol. 2016 Feb 3;36:5B.4.1-10. doi: 10.1002/9780470151808.sc05b04s36. Review.

PMID:
26840227
48.

Impulse oscillometry identifies peripheral airway dysfunction in children with adenosine deaminase deficiency.

Komarow HD, Sokolic R, Hershfield MS, Kohn DB, Young M, Metcalfe DD, Candotti F.

Orphanet J Rare Dis. 2015 Dec 18;10:159. doi: 10.1186/s13023-015-0365-z.

49.
50.

Gene therapy outpaces haplo for SCID-X1.

Kohn DB.

Blood. 2015 Jun 4;125(23):3521-2. doi: 10.1182/blood-2015-04-641720.

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