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Items: 34

1.

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

Maeder ML, Stefanidakis M, Wilson CJ, Baral R, Barrera LA, Bounoutas GS, Bumcrot D, Chao H, Ciulla DM, DaSilva JA, Dass A, Dhanapal V, Fennell TJ, Friedland AE, Giannoukos G, Gloskowski SW, Glucksmann A, Gotta GM, Jayaram H, Haskett SJ, Hopkins B, Horng JE, Joshi S, Marco E, Mepani R, Reyon D, Ta T, Tabbaa DG, Samuelsson SJ, Shen S, Skor MN, Stetkiewicz P, Wang T, Yudkoff C, Myer VE, Albright CF, Jiang H.

Nat Med. 2019 Feb;25(2):229-233. doi: 10.1038/s41591-018-0327-9. Epub 2019 Jan 21.

PMID:
30664785
2.

Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice.

Shen S, Sanchez ME, Blomenkamp K, Corcoran EM, Marco E, Yudkoff CJ, Jiang H, Teckman JH, Bumcrot D, Albright CF.

Hum Gene Ther. 2018 Aug;29(8):861-873. doi: 10.1089/hum.2017.227. Epub 2018 Jun 22.

PMID:
29641323
3.

Continuous intranigral infusion is not associated with observable behavioral deficits or marked pathology: a preclinical safety study.

Grondin R, Ai Y, Hardy PA, Butt MT, Nelson BD, Lemmon JD, Bumcrot D, Gash DM, Gerhardt GA, Zhang Z.

J Neurosurg. 2017 Apr;126(4):1253-1262. doi: 10.3171/2016.2.JNS152295. Epub 2016 May 27.

PMID:
27231974
4.

Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications.

Friedland AE, Baral R, Singhal P, Loveluck K, Shen S, Sanchez M, Marco E, Gotta GM, Maeder ML, Kennedy EM, Kornepati AV, Sousa A, Collins MA, Jayaram H, Cullen BR, Bumcrot D.

Genome Biol. 2015 Nov 24;16:257. doi: 10.1186/s13059-015-0817-8.

5.

Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.

Gori JL, Hsu PD, Maeder ML, Shen S, Welstead GG, Bumcrot D.

Hum Gene Ther. 2015 Jul;26(7):443-51. doi: 10.1089/hum.2015.074. Review.

PMID:
26068008
6.

Tissue-specific gene silencing monitored in circulating RNA.

Sehgal A, Chen Q, Gibbings D, Sah DW, Bumcrot D.

RNA. 2014 Feb;20(2):143-9. doi: 10.1261/rna.042507.113. Epub 2013 Dec 19.

7.

First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement.

Tabernero J, Shapiro GI, LoRusso PM, Cervantes A, Schwartz GK, Weiss GJ, Paz-Ares L, Cho DC, Infante JR, Alsina M, Gounder MM, Falzone R, Harrop J, White AC, Toudjarska I, Bumcrot D, Meyers RE, Hinkle G, Svrzikapa N, Hutabarat RM, Clausen VA, Cehelsky J, Nochur SV, Gamba-Vitalo C, Vaishnaw AK, Sah DW, Gollob JA, Burris HA 3rd.

Cancer Discov. 2013 Apr;3(4):406-17. doi: 10.1158/2159-8290.CD-12-0429. Epub 2013 Jan 28.

8.

An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe(-/-) mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia.

Schmidt PJ, Toudjarska I, Sendamarai AK, Racie T, Milstein S, Bettencourt BR, Hettinger J, Bumcrot D, Fleming MD.

Blood. 2013 Feb 14;121(7):1200-8. doi: 10.1182/blood-2012-09-453977. Epub 2012 Dec 6.

9.

Alpha-synuclein suppression by targeted small interfering RNA in the primate substantia nigra.

McCormack AL, Mak SK, Henderson JM, Bumcrot D, Farrer MJ, Di Monte DA.

PLoS One. 2010 Aug 11;5(8):e12122. doi: 10.1371/journal.pone.0012122.

10.

Claudin-3 gene silencing with siRNA suppresses ovarian tumor growth and metastasis.

Huang YH, Bao Y, Peng W, Goldberg M, Love K, Bumcrot DA, Cole G, Langer R, Anderson DG, Sawicki JA.

Proc Natl Acad Sci U S A. 2009 Mar 3;106(9):3426-30. doi: 10.1073/pnas.0813348106. Epub 2009 Feb 10.

11.

In vivo silencing of alpha-synuclein using naked siRNA.

Lewis J, Melrose H, Bumcrot D, Hope A, Zehr C, Lincoln S, Braithwaite A, He Z, Ogholikhan S, Hinkle K, Kent C, Toudjarska I, Charisse K, Braich R, Pandey RK, Heckman M, Maraganore DM, Crook J, Farrer MJ.

Mol Neurodegener. 2008 Nov 1;3:19. doi: 10.1186/1750-1326-3-19.

12.

Assessing siRNA pharmacodynamics in a luciferase-expressing mouse.

Svensson RU, Shey MR, Ballas ZK, Dorkin JR, Goldberg M, Akinc A, Langer R, Anderson DG, Bumcrot D, Henry MD.

Mol Ther. 2008 Dec;16(12):1995-2001. doi: 10.1038/mt.2008.187. Epub 2008 Sep 9.

13.

MicroRNA-34 mediates AR-dependent p53-induced apoptosis in prostate cancer.

Rokhlin OW, Scheinker VS, Taghiyev AF, Bumcrot D, Glover RA, Cohen MB.

Cancer Biol Ther. 2008 Aug;7(8):1288-96. Epub 2008 Aug 13.

PMID:
18497571
14.

Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway.

John M, Constien R, Akinc A, Goldberg M, Moon YA, Spranger M, Hadwiger P, Soutschek J, Vornlocher HP, Manoharan M, Stoffel M, Langer R, Anderson DG, Horton JD, Koteliansky V, Bumcrot D.

Nature. 2007 Oct 11;449(7163):745-7. Epub 2007 Sep 26.

15.

Calcium/calmodulin-dependent kinase II plays an important role in prostate cancer cell survival.

Rokhlin OW, Taghiyev AF, Bayer KU, Bumcrot D, Koteliansk VE, Glover RA, Cohen MB.

Cancer Biol Ther. 2007 May;6(5):732-42. Epub 2007 Feb 5.

PMID:
17387273
16.

RNAi therapeutics: a potential new class of pharmaceutical drugs.

Bumcrot D, Manoharan M, Koteliansky V, Sah DW.

Nat Chem Biol. 2006 Dec;2(12):711-9. Review.

PMID:
17108989
17.

RNAi-mediated gene silencing in non-human primates.

Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, Harborth J, Heyes JA, Jeffs LB, John M, Judge AD, Lam K, McClintock K, Nechev LV, Palmer LR, Racie T, Röhl I, Seiffert S, Shanmugam S, Sood V, Soutschek J, Toudjarska I, Wheat AJ, Yaworski E, Zedalis W, Koteliansky V, Manoharan M, Vornlocher HP, MacLachlan I.

Nature. 2006 May 4;441(7089):111-4. Epub 2006 Mar 26.

PMID:
16565705
18.

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs.

Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP.

Nature. 2004 Nov 11;432(7014):173-8.

19.

Small-molecule modulators of Hedgehog signaling: identification and characterization of Smoothened agonists and antagonists.

Frank-Kamenetsky M, Zhang XM, Bottega S, Guicherit O, Wichterle H, Dudek H, Bumcrot D, Wang FY, Jones S, Shulok J, Rubin LL, Porter JA.

J Biol. 2002 Nov 6;1(2):10.

20.

Comparative biological responses to human Sonic, Indian, and Desert hedgehog.

Pathi S, Pagan-Westphal S, Baker DP, Garber EA, Rayhorn P, Bumcrot D, Tabin CJ, Blake Pepinsky R, Williams KP.

Mech Dev. 2001 Aug;106(1-2):107-17.

21.

Relationship between dose, distance and time in Sonic Hedgehog-mediated regulation of anteroposterior polarity in the chick limb.

Yang Y, Drossopoulou G, Chuang PT, Duprez D, Marti E, Bumcrot D, Vargesson N, Clarke J, Niswander L, McMahon A, Tickle C.

Development. 1997 Nov;124(21):4393-404.

22.

Sonic hedgehog promotes the survival of specific CNS neuron populations and protects these cells from toxic insult In vitro.

Miao N, Wang M, Ott JA, D'Alessandro JS, Woolf TM, Bumcrot DA, Mahanthappa NK, Pang K.

J Neurosci. 1997 Aug 1;17(15):5891-9.

23.

A role for Indian hedgehog in extraembryonic endoderm differentiation in F9 cells and the early mouse embryo.

Becker S, Wang ZJ, Massey H, Arauz A, Labosky P, Hammerschmidt M, St-Jacques B, Bumcrot D, McMahon A, Grabel L.

Dev Biol. 1997 Jul 15;187(2):298-310.

24.

Sonic hedgehog: making the gradient.

Bumcrot DA, McMahon AP.

Chem Biol. 1996 Jan;3(1):13-6. Review.

25.

Combinatorial signaling by Sonic hedgehog and Wnt family members induces myogenic bHLH gene expression in the somite.

Münsterberg AE, Kitajewski J, Bumcrot DA, McMahon AP, Lassar AB.

Genes Dev. 1995 Dec 1;9(23):2911-22.

26.

Induction of dopaminergic neuron phenotype in the midbrain by Sonic hedgehog protein.

Wang MZ, Jin P, Bumcrot DA, Marigo V, McMahon AP, Wang EA, Woolf T, Pang K.

Nat Med. 1995 Nov;1(11):1184-8.

PMID:
7584992
27.

Distribution of Sonic hedgehog peptides in the developing chick and mouse embryo.

Martí E, Takada R, Bumcrot DA, Sasaki H, McMahon AP.

Development. 1995 Aug;121(8):2537-47.

28.

Somite differentiation. Sonic signals somites.

Bumcrot DA, McMahon AP.

Curr Biol. 1995 Jun 1;5(6):612-4. Review.

29.

Requirement of 19K form of Sonic hedgehog for induction of distinct ventral cell types in CNS explants.

Martí E, Bumcrot DA, Takada R, McMahon AP.

Nature. 1995 May 25;375(6529):322-5.

PMID:
7753196
30.

Proteolytic processing yields two secreted forms of sonic hedgehog.

Bumcrot DA, Takada R, McMahon AP.

Mol Cell Biol. 1995 Apr;15(4):2294-303. Erratum in: Mol Cell Biol 1995 May;15(5):2904.

31.
32.

In vitro analysis of germ cell genotoxicity in testis explant cultures: spermatid micronucleus assays.

Risley MS, Miller A, Bumcrot DA.

Mutat Res. 1988 Apr;203(2):125-33.

PMID:
3127703
33.

In vitro maintenance of spermatogenesis in Xenopus laevis testis explants cultured in serum-free media.

Risley MS, Miller A, Bumcrot DA.

Biol Reprod. 1987 May;36(4):985-97.

PMID:
3593863
34.

Changes in DNA topology during spermatogenesis.

Risley MS, Einheber S, Bumcrot DA.

Chromosoma. 1986;94(3):217-27.

PMID:
3490360

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