Kleinstiver Lab Publications

​Kleinstiver lab members in bold text.
* co-contributing authors / ^ co-corresponding authors

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Published manuscripts:​​

1. Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice.
   Alves CRR*, Das S*, Krishnan V*, Ha LL**, Fox LR**, Stutzman HE**, Shamber CE**, Kalailingam P**, McCarthy S**, Lino Cardenas CL**, Fong CE, Imai T, Mitra S, Yun S, Wood RK, Benning FMC, Roh K, Lawton J, Kim N, Silverstein RA, Ferreira da Silva J, de la Cruz D, Richa R, Xie J, Gray-Edwards HL, Malhotra R, Chung DY, Chao LH, Tsai SQ, Maguire CA, Lindsay ME^, Kleinstiver BP^, Musolino PL^. Nature Biomedical Engineering. (2025)

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2. GenomePAM directs PAM characterization and engineering of CRISPR-Cas nucleases using mammalian genome repeats.
   Yu M, Ai L, Wang B, Lian S, Ip L, Liu J, Li L, Tsai SQ, Kleinstiver BP, Zheng Z. Nature Biomedical Engineering. (2025)
    

3. An opportune time for targeted brain arteriovenous malformation therapy
   Hale AT, Kalailingam P, Kundishora AJ, Reeves BC, Brastianos PK, Shih HA, Jain RK, Ning M, Stapleton CJ, Patel AB, Smith ER, Chapman PH, Rabinov JD, Kleinstiver BP, Musolino PL, Kahle KT. Journal of Neurosurgery. (2025)
    

4. Engineering of CD63 Enables Selective Extracellular Vesicle Cargo Loading and Enhanced Payload Delivery.
   Obuchi W, Zargani-Piccardi A, Leandro K, Rufino-Ramos D, Di Lanni E, Frederick DM, Maalouf K, Nieland L, Xiao T, Repiton P, Vaine CA, Kleinstiver BP, Bragg DC, Lee H, Miller MA, Breakefield XO, Breyne K. Journal of Extracellular Vesicles. (2025)
    

5. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
   Musunuru K*,^ Grandinette SA*, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC^.  The New England Journal of Medicine. (2025)
    

6. Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning.
   Silverstein RA, Kim N*, Kroell AS*, Walton RT, Delano J, Butcher RM, Pacesa M, Smith BK, Christie KA, Ha LL, Meis RJ, Clark AB, Spinner AD, Lazzarotto CR, Li Y, Matsubara A, Urbina EO, Dahl GA, Correia BE, Marks DS, Tsai SQ, Pinello L, De Ravin SS, Liu Q, Kleinstiver BP.  Nature. (2025)

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7. Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of TOR1A DYT1 allele in a xenograft mouse model.
   Maalouf KE, Frederick DM, Sharma N, Haidar EA, Xiao T, Han JS, Mahamdeh MS, Soberman RJ, Rufino-Ramos D, Kleinstiver BP, Jinnah HA, Vaine CA, Bragg DC, Breyne K.  Molecular Therapy Nucleic Acids. (2025)
    

8. CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice.
   Nieland L, Vrijmoet AB, Jetten IW, Rufino-Ramos D, de Reus AJEM, Breyne K, Kleinstiver BP, Maguire CA, Broekman MLD, Breakefield XO, Abels ER. Molecular Therapy. (2024)
    

9. Environmental Impact, Cost, and Acceptability of a Laboratory Sustainability Certification Program for Biomedical Research in an Academic Medical Center. 
   Padget M, Sörme P, Spezia M, Slaugenhaupt SA, McKowen J, Kirchner E, Ameral N, Reis SA, Slater D, Haggarty, SJ, Kleinstiver BP, Dunn EC, Larocque R, Alves CRR, Stern M, Pelli A, Duhaime AC. The Journal of Climate Change and Health. (2024)
    

10. Myocardial infarction augments sleep to limit cardiac inflammation and damage.
    Huynh P, Hoffmann JD, Gerhardt T, Kiss MG, Zuraikat FM, Cohen O, Wolfram C, Yates AG, Leunig A, Heiser M, Gaebel L, Gianeselli M, Goswami S, Khamhoung A, Downey J, Yoon S, Chen Z, Roudko V, Dawson T, Ferreira da Silva J, Ameral NJ, Morgenroth-Rebin J, D'Souza D, Koekkoek LL, Jacob W, Munitz J, Lee D, Fullard JF, van Leent MMT, Roussos P, Kim-Schulze S, Shah N, Kleinstiver BP, Swirski FK, Leistner D, St-Onge MP, McAlpine CS. Nature. (2024)
     

11. High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease. 
    Bzhilyanskaya V*, Ma L*, Liu S, Fox LR, Whittaker MN, Meis RJ, Choi U, Lawson A, Ma M, Theobald N, Burkett S, Sweeney CL, Lazzarotto CR, Tsai SQ, Lack JB, Wu X, Dahl GA, Malech HL, Kleinstiver BP^, De Ravin SS^. Science Translational Medicine. (2024)
     

12. Deciphering the impact of genomic variation on function.
    IGVF Consortium (incl. Kim N, King EM, & Kleinstiver BP).  Nature. (2024)
     

13. Click editing enables programmable genome writing using DNA polymerases and HUH endonucleases.
    Ferreira da Silva J*, Tou CJ*, King EM, Eller ML, Rufino-Ramos D, Ma L, Cromwell CR, Metovic J, Benning FMC, Chao LH, Eichler FS, Kleinstiver BP. Nature Biotechnology. (2024)

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14. Programmable RNA-guided enzymes for next-generation genome editing.
    Tou CJ, Kleinstiver BP. Nature. (2024)
     

15. Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads.
    Leandro K, Rufino-Ramos D, Breyne K, Di Ianni E, Lopes SM, Jorge Nobre R, Kleinstiver BP, Perdigão PRL, Breakefield XO, Pereira de Almeida L. Advanced Drug Delivery Reviews. (2024)
     

16. CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics.
    Mesaki K, Yamamoto H, Juvet S, Yeung J, Guan Z, Akhter A, Yao Y, Dickie C, Mangat H, Wang A, Wilson GW, Mariscal A, Hu J, Davidson AR, Kleinstiver BP, Cypel M, Liu M, Keshavjee S. Human Gene Therapy. (2024)
     

17. Splice modulators target PMS1 to reduce somatic expansion of the Huntington's disease-associated CAG repeat.
    McLean ZL, Gao D, Correia K, Roy JCL, Shibata S, Farnum IN, Valdepenas-Mellor Z, Kovalenko M, Rapuru M, Morini E, Ruliera J, Gillis T, Lucente D, Kleinstiver BP, Lee JM, MacDonald ME, Wheeler VC, Mouro Pinto R, Gusella JF.  Nature Communications. (2024)
     

18. In situ architecture of Opa1-dependent mitochondrial cristae remodeling.
    Fry MY, Navarro PP, Hakim P, Ananda VY, Qin X, Landoni JC, Rath S, Inde Z, Lugo CM, Luce BE, Ge Y, McDonald JL, Ali I, Ha LL, Kleinstiver BP, Chan DC, Sarosiek KA, Chao LH. EMBO Journal. (2024)
     

19. Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy.
    Alves CRR^, Ha LL, Yaworski R, Sutton ER, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP.Nature Biomedical Engineering. (2023)

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20. PAM-flexible genome editing with an engineered chimeric Cas9.
    Zhao L, Koseki SRT, Silverstein RA, Amrani N, Peng C, Kramme C, Savic N, Pacesa M, Rodríguez TC, Stan T, Tysinger E, Hong L, Yudistyra V, Ponnapati MR, Jacobson JM, Church GM, Jakimo N, Truant R, Jinek M, Kleinstiver BP, Sontheimer EJ, Chatterjee P. Nature Communications. (2023)
     

21. Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo.
    Tao Y, Lamas V, Du W, Zhu W, Li Y, Whittaker MN, Zuris JA, Thompson DB, Rameshbabu AP, Shu Y, Gao X, Hu JH, Pei C, Kong WJ, Liu X, Wu H, Kleinstiver BP, Liu DR, Chen ZY. Nature Communications. (2023)
     

22. Immunomodulation of the donor lung with CRISPR-mediated activation of IL-10 expression. 
    Mesaki K, Juvet S, Yeung J, Guan Z, Wilson GW, Hu J, Davidson AR, Kleinstiver BP, Cypel M, Liu M, Keshavjee S. Journal of Heart and Lung Transplantation. (2023)
     

23. Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F.
    Peters CW, Hanlon KS, Ivanchenko MV, Zinn E, Linarte EF, Li Y, Levy JM, Liu DR, Kleinstiver BP, Indzhykulian AA, Corey DP. Molecular Therapy. (2023)
     

24. Interleukin-3 coordinates glial-peripheral immune crosstalk to incite multiple sclerosis.
    Kiss MG, Mindur JE, Yates AG, Lee D, Fullard JF, Anzai A, Poller WC, Christie KA, Iwamoto Y, Roudko V, Downey J, Chan CT, Huynh P, Janssen H, Ntranos A, Hoffmann JD, Jacob W, Goswami S, Singh S, Leppert D, Kuhle J, Kim-Schulze S, Nahrendorf M, Kleinstiver BP, Probert F, Roussos P, Swirski FK, McAlpine CS. Immunity. (2023)
     

25. Multimodal CRISPR perturbations of GWAS loci associated with coronary artery disease in vascular endothelial cells.
    Wünnemann F, Fotsing Tadjo T, Beaudoin M, Lalonde S, Lo KS, Kleinstiver BP, Lettre G. PLoS Genetics. (2023)
     

26. Sniper2L is a high-fidelity Cas9 variant with high activity.
    Kim YH, Kim N, Okafor I, Choi S, Min S, Lee J, Bae SM, Choi K, Choi J, Harihar V, Kim Y, Kim JS, Kleinstiver BP, Lee JK, Ha T, Kim HH. Nature Chemical Biology. (2023)
     

27. SPTSSA variants alter sphingolipid synthesis and cause a complex hereditary spastic paraplegia.
    Srivastava S, Shaked HM, Gable K, Gupta SD, Pan X, Somashekarappa N, Han G, Mohassel P, Gotkine M, Doney E, Goldenberg P, Tan QKG, Gong Y, Kleinstiver BP, Wishart B, Cope H, Pires CB, Stutzman HE, Spillmann RC; Undiagnosed Disease Network; Sadjadi R, Elpeleg O, Lee CH, Bellen HJ, Edvardson S, Eichler F, Dunn TM. Brain. (2023)
     

28. CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells. 
    Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Frontiers in Immunology. (2023)
     

29. Precise cut-and-paste DNA insertion using engineered type V-K CRISPR-associated transposases. 
    Tou CJ, Orr B, Kleinstiver BP. Nature Biotechnology. (2023)

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30. Evolution of CRISPR-associated endonucleases as inferred from resurrected proteins.
    Alonso-Lerma B, Jabalera Y, Samperio S, Morin M, Fernandez A, Hille LT, Silverstein RA, Quesada-Ganuza A, Reifs A, Fernández-Peñalver S, Benitez Y, Soletto L, Gavira JA, Diaz A, Vranken W, Sanchez-Mejias A, Güell M, Mojica FJM, Kleinstiver BP, Moreno-Pelayo MA, Montoliu L, Perez-Jimenez R.Nature Microbiology. (2023)
     

31. Expanding the Scope of Bacterial CRISPR Activation with PAM-Flexible dCas9 Variants.
    Kiattisewee C, Karanjia AV, Legut M, Daniloski Z, Koplik SE, Nelson J, Kleinstiver BP, Sanjana NE, Carothers JM, Zalatan JG.ACS Synthetic Biology. (2022)
     

32. Precise DNA cleavage using CRISPR-SpRYgests. 
    Christie KA, Guo JA, Silverstein RA, Doll RA, Mabuchi M, Stutzman HE, Lin J, Ma L, Walton RT, Pinello L, Robb GB, Kleinstiver BP. Nature Biotechnology. (2022)

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33. Recent Advances in Double-Strand Break-Free Kilobase-Scale Genome Editing Technologies.
    Tou CJ, Kleinstiver BP. Biochemistry. (2022)
     

34. Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR-Cas9.
    Shin JW, Hong EP, Park SS, Choi DE, Seong IS, Whittaker MN, Kleinstiver BP, Chen RZ, Lee JM. JCI Insight. (2022)
     

35. Lack of Cas13a inhibition by anti-CRISPR proteins from Leptotrichia prophages.
    Meeske AJ, Johnson MC, Hille LT, Kleinstiver BP, Bondy-Denomy J.Molecular Cell. (2022)​
     

36. Genome editing in animals with minimal PAM CRISPR-Cas9 enzymes.
    Vicencio J, Sánchez-Bolaños C, Moreno-Sánchez I, Brena D, Kukhtar D, Ruiz-López M, Cots-Ponjoan M, Vejnar CE, Rubio A, Rodrigo Melero N, Carolis C, Pérez-Pulido AJ, Giráldez AJ, Kleinstiver BP, Cerón J, Moreno-Mateos MA. Nature Communications. (2022)​​
     

37. CRISPR-Cas knockout of miR21 reduces glioma growth.
    Nieland L, van Solinge TS, Cheah PS, Morsett LM, El Khoury J, Rissman JI, Kleinstiver BP, Broekman MLD, Breakefield XO, Abels ER. Molecular Therapy Oncolytics. (2022)
     

38. Making the cut with PAMless CRISPR-Cas enzymes.
    Christie KA, Kleinstiver BP. Trends in Genetics. (2021)
     

39. Astrocytic interleukin-3 programs microglia and limits Alzheimer's disease.
    McAlpine CS, Park J, Griciuc A, Kim E, Choi SH, Iwamoto Y, Kiss MG, Christie KA, Vinegoni C, Poller WC, Mindur JE, Chan CT, He S, Janssen H, Wong LP, Downey J, Singh S, Anzai A, Kahles F, Jorfi M, Feruglio PF, Sadreyev RI, Weissleder R, Kleinstiver BP, Nahrendorf M, Tanzi RE, Swirski FK. Nature. (2021) 
     

40. NNT mediates redox-dependent pigmentation via a UVB- and MITF-independent mechanism.
    Allouche J, Rachmin I, Adhikari K, Pardo LM, Lee JH, McConnell AM, Kato S, Fan S, Kawakami A, Suita Y, Wakamatsu K, Igras V, Zhang J, Navarro PP, Lugo CM, Noonan HR, Christie KA, Itin K, Mujahid N, Lo JA, Won CH, Evans CL, Weng QY, Wang H, Osseiran S, Lovas A, Németh I, Cozzio A, Navarini AA, Hsiao JJ, Nguyen N, Kemény LV, Iliopoulos O, Berking C, Ruzicka T, Gonzalez-José R, Bortolini MC, Canizales-Quinteros S, Acuna-Alonso V, Gallo C, Poletti G, Bedoya G, Rothhammer F, Ito S, Schiaffino MV, Chao LH, Kleinstiver BP, Tishkoff S, Zon LI, Nijsten T, Ruiz-Linares A, Fisher DE, Roider E. Cell. (2021)
     

41. Whole-genome sequencing association analysis of quantitative red blood cell phenotypes: The NHLBI TOPMed program.
    Hu Y, Stilp AM, McHugh CP, Rao S, Jain D, Zheng X, Lane J, Méric de Bellefon S, Raffield LM, Chen MH, Yanek LR, Wheeler M, Yao Y, Ren C, Broome J, Moon JY, de Vries PS, Hobbs BD, Sun Q, Surendran P, Brody JA, Blackwell TW, Choquet H, Ryan K, Duggirala R, Heard-Costa N, Wang Z, Chami N, Preuss MH, Min N, Ekunwe L, Lange LA, Cushman M, Faraday N, Curran JE, Almasy L, Kundu K, Smith AV, Gabriel S, Rotter JI, Fornage M, Lloyd-Jones DM, Vasan RS, Smith NL, North KE, Boerwinkle E, Becker LC, Lewis JP, Abecasis GR, Hou L, O'Connell JR, Morrison AC, Beaty TH, Kaplan R, Correa A, Blangero J, Jorgenson E, Psaty BM, Kooperberg C, Walton RT, Kleinstiver BP, Tang H, Loos RJF, Soranzo N, Butterworth AS, Nickerson D, Rich SS, Mitchell BD, Johnson AD, Auer PL, Li Y, Mathias RA, Lettre G, Pankratz N, Laurie CC, Laurie CA, Bauer DE, Conomos MP, Reiner AP; NHLBI Trans-Omics for Precision Medicine (TOPMed) Consortium. American Journal of Human Genetics. (2021)
     

42. CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
    Brault J, Liu TQ, Bello EA, Liu S, Sweeney CL, Meis RJ, Koontz S, Corsino C, Choi U, Vayssière G, Bosticardo M, Dowdell KC, Lazzarotto CR, Clark A, Notarangelo LD, Ravell JC, Lenardo MJ, Kleinstiver BP, Tsai S, Wu X, Dahl GA, Malech HL, De Ravin SS. Blood. (2021)
     

43. Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells.
    De Ravin SS, Brault J, Meis RJ, Liu S, Li L, Pavel-Dinu M, Lazzarotto CR, Liu T, Koontz SM, Choi U, Sweeney CL, Theobald N, Lee G, Clark AB, Burkett SS, Kleinstiver BP, Porteus MH, Tsai S, Kuhns DB, Dahl GA, Headey S, Wu X, Malech HL. Blood. (2021)
     

44. Scalable characterization of the PAM requirements of CRISPR-Cas enzymes using HT-PAMDA.
    Walton RT, Hsu JY, Joung JK, Kleinstiver BP. Nature Protocols. (2021)
     

45. Plant genome editing branches out.
    Hille LT, Kleinstiver BP. Nature Plants. (2021)
     

46. enAsCas12a Enables CRISPR-Directed Evolution to Screen for Functional Drug Resistance Mutations in Sequences Inaccessible to SpCas9.
    Neggers JE, Jacquemyn M, Dierckx T, Kleinstiver BP, Thibaut HJ, Daelemans D. Molecular Therapy (2021)
     

47. Optimization of AsCas12a for combinatorial genetic screens in human cells.
    DeWeirdt PC, Sanson KR, Sangree AK, Hegde M, Hanna RE, Feeley MN, Griffith AL, Teng T, Borys SM, Strand C, Joung JK, Kleinstiver BP, Pan X, Huang A, Doench JG. Nature Biotechnology. (2021) 
     

48. Cell-based artificial APC resistant to lentiviral transduction for efficient generation of CAR-T cells from various cell sources.
    Schmidts A, Marsh LC, Srivastava AA, Bouffard AA, Boroughs AC, Scarfò I, Larson RC, Bedoya F, Choi BD, Frigault MJ, Bailey SR, Leick MB, Vatsa S, Kann MC, Prew MS, Kleinstiver BP, Joung JK, Maus MV. Journal for ImmunoTherapy of Cancer. (2020)
     

49. Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function.
    Cruz L, György B, Cheah PS, Kleinstiver BP, Eimer WA, Garcia SP, Sharma N, Ozelius LJ, Bragg DC, Joung JK, Norberto de Souza O, Macedo Timmers LFS, Breakefield XO. Molecular Therapy Nucleic Acids. (2020)
     

50. Listeria Phages Induce Cas9 Degradation to Protect Lysogenic Genomes.
    Osuna BA, Karambelkar S, Mahendra C, Christie KA, Garcia B, Davidson AR, Kleinstiver BP, Kilcher S, Bondy-Denomy J. Cell Host Microbe. (2020)
     

51. Broad-spectrum anti-CRISPR proteins facilitate horizontal gene transfer.
    Mahendra C, Christie KA, Osuna BA, Pinilla-Redondo R, Kleinstiver BP, Bondy-Denomy J. Nature Microbiology. (2020)
     

52. Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants.
    Walton RT, Christie KA, Whittaker MN, Kleinstiver BP. Science. (2020)
     

53. In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery.
    Breuer CB, Hanlon KS, Natasan JS, Volak A, Meliani A, Mingozzi F, Kleinstiver BP, Moon JJ, Maguire CA. Scientific Reports. (2020)
     

54. High levels of AAV vector integration into CRISPR-induced DNA breaks.
    Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Lööv C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, György B. Nature Communications. (2019)
     

55. Voices in methods development.
    Anikeeva P, Boyden E, Brangwynne C, Cissé II, Fiehn O, Fromme P, Gingras AC, Greene CS, Heard E, Hell SW, Hillman E, Jensen GJ, Karchin R, Kiessling LL, Kleinstiver BP, Knight R, Kukura P, Lancaster MA, Loman N, Looger L, Lundberg E, Luo Q, Miyawaki A, Myers EW Jr, Nolan GP, Picotti P, Reik W, Sauer M, Shalek AK, Shendure J, Slavov N, Tanay A, Troyanskaya O, van Valen D, Wang HW, Yi C, Yin P, Zernicka-Goetz M, Zhuang X. Nature Methods. (2019)
     

56. Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss.
    György B, Nist-Lund C, Pan B, Asai Y, Karavitaki KD, Kleinstiver BP, Garcia SP, Zaborowski MP, Solanes P, Spataro S, Schneider BL, Joung JK, Géléoc GSG, Holt JR, Corey DP. Nature Medicine. (2019)
     

57. Engineered CRISPR-Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing.
    Kleinstiver BP^, Sousa AA, Walton RT, Tak YE, Hsu JY, Clement K, Welch MM, Horng JE, Malagon-Lopez J, Scarfò I, Maus MV, Pinello L, Aryee MJ, Joung JK. Nature Biotechnology. (2019)
     

58. Activities and specificities of CRISPR/Cas9 and Cas12a nucleases for targeted mutagenesis in maize.
    Lee K, Zhang Y, Kleinstiver BP, Guo JA, Aryee MJ, Miller J, Malzahn A, Zarecor S, Lawrence-Dill CJ, Joung JK, Qi Y, Wang K. Plant Biotechnology Journal. (2019)
     

59. Discovery of widespread type I and type V CRISPR-Cas inhibitors.
    Marino ND, Zhang JY, Borges AL, Sousa AA, Leon LM, Rauch BJ, Walton RT, Berry JD, Joung JK, Kleinstiver BP, Bondy-Denomy J. Science. (2018)
     

60. Prediction of off-target activities for the end-to-end design of CRISPR guide RNAs.
    Listgarten J, Weinstein M, Kleinstiver BP, Sousa AA, Joung JK, Crawford J, Gao K, Hoang L, Elibol M, Doench JG, Fusi N. Nature Biomedical Engineering. (2018)
     

61. CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease.
    György B, Lööv C, Zaborowski MP, Takeda S, Kleinstiver BP, Commins C, Kastanenka K, Mu D, Volak A, Giedraitis V, Lannfelt L, Maguire CA, Joung JK, Hyman BT, Breakefield XO, Ingelsson M. Molecular Therapy Nucleic Acids. (2018)
     

62. Temporal and Spatial Post-Transcriptional Regulation of Zebrafish tie1 mRNA by Long Noncoding RNA During Brain Vascular Assembly.
    Chowdhury TA, Koceja C, Eisa-Beygi S, Kleinstiver BP, Kumar SN, Lin CW, Li K, Prabhudesai S, Joung JK, Ramchandran R. Arteriosclerosis, Thrombosis, and Vascular Biology. (2018)
     

63. Allele-Specific CRISPR-Cas9 Genome Editing of the Single-Base P23H Mutation for Rhodopsin-Associated Dominant Retinitis Pigmentosa.
    Li P, Kleinstiver BP, Leon MY, Prew MS, Navarro-Gomez D, Greenwald SH, Pierce EA, Joung JK, Liu Q. The CRISPR Journal (2018)
     

64. Camptothecin resistance is determined by the regulation of topoisomerase I degradation mediated by ubiquitin proteasome pathway.
    Ando K, Shah AK, Sachdev V, Kleinstiver BP, Taylor-Parker J, Welch MM, Hu Y, Salgia R, White FM, Parvin JD, Ozonoff A, Rameh LE, Joung JK, Bharti AK. Oncotarget. (2017)
     

65. Inducible and multiplex gene regulation using CRISPR-Cpf1-based transcription factors.
    Tak YE, Kleinstiver BP, Nuñez JK, Hsu JY, Horng JE, Gong J, Weissman JS, Joung JK. Nature Methods. (2017)
     

66. Enhanced proofreading governs CRISPR-Cas9 targeting accuracy.
    Chen JS*, Dagdas YS*, Kleinstiver BP*, Welch MM, Sousa AA, Harrington LB, Sternberg SH, Joung JK, Yildiz A, Doudna JA. Nature. (2017)
     

67. Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells.
    Kleinstiver BP*^, Tsai SQ*, Prew MS, Nguyen NT, Welch MM, Lopez JM, McCaw ZR, Aryee MJ, Joung JK.Nature Biotechnology. (2016)
     

68. Isocitrate Dehydrogenase Mutations Confer Dasatinib Hypersensitivity and SRC Dependence in Intrahepatic Cholangiocarcinoma.
    Saha SK, Gordan JD, Kleinstiver BP, Vu P, Najem MS, Yeo JC, Shi L, Kato Y, Levin RS, Webber JT, Damon LJ, Egan RK, Greninger P, McDermott U, Garnett MJ, Jenkins RL, Rieger-Christ KM, Sullivan TB, Hezel AF, Liss AS, Mizukami Y, Goyal L, Ferrone CR, Zhu AX, Joung JK, Shokat KM, Benes CH, Bardeesy N. Cancer Discovery. (2016)
     

69. High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.
    Kleinstiver BP*, Pattanayak V*, Prew MS, Tsai SQ, Nguyen NT, Zheng Z, Joung JK. Nature. (2016) 
     

70. Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition.
    Kleinstiver BP^, Prew MS, Tsai SQ, Nguyen NT, Topkar VV, Zheng Z, Joung JK. Nature Biotechnology. (2015)
     

71. Engineered CRISPR-Cas9 nucleases with altered PAM specificities.
    Kleinstiver BP, Prew MS, Tsai SQ, Topkar VV, Nguyen NT, Zheng Z, Gonzales AP, Li Z, Peterson RT, Yeh JR, Aryee MJ, Joung JK. Nature. (2015)
     

72. Hypoxia drives transient site- specific copy gain and drug-resistant gene expression.
    Black JC, Atabakhsh E, Kim J, Biette KM, Van Rechem C, Ladd B, Burrowes PD, Donado C, Mattoo H, Kleinstiver BP, Song B, Andriani G, Joung JK, Iliopoulos O, Montagna C, Pillai S, Getz G, Whetstine JR. Genes and Development. (2015)
     

73. The I-TevI nuclease and linker domains contribute to the specificity of monomeric TALENs.
    Kleinstiver BP*, Wang L*, Wolfs JM, Kolaczyk T, McDowell B, Wang X, Schild-Poulter C, Bogdanove AJ, Edgell DR.G3. (2014)
     

74. Rapid screening of endonuclease target site preference using a modified bacterial two-plasmid selection.
    Wolfs JM, Kleinstiver BP, Edgell DR. Methods of Molecular Biology. (2014)
     

75. The monomeric GIY-YIG homing endonuclease I-BmoI uses a molecular anchor and a flexible tether to sequentially nick DNA.
    Kleinstiver BP, Wolfs JM, Edgell DR. Nucleic Acids Research. (2013) 
     

76. Monomeric site-specific nucleases for genome editing.
    Kleinstiver BP, Wolfs JM, Kolaczyk T, Roberts AK, Hu SX, Edgell DR. Proceedings of the National Academy of Science USA. (2012)
     

77. Divalent metal ion differentially regulates the sequential nicking reactions of the GIY-YIG homing endonuclease I-BmoI.
    Kleinstiver BP, Bérubé-Janzen W, Fernandes AD, Edgell DR. PLoS One. (2011)
     

78. Estimating the evidence of selection and the reliability of inference in unigenic evolution.
    Fernandes AD, Kleinstiver BP, Edgell DR, Wahl LM, Gloor GB. Algorithms of Molecular Biology. (2010) 
     

79. A unified genetic, computational and experimental framework identifies functionally relevant residues of the homing endonuclease I-BmoI.
    Kleinstiver BP, Fernandes AD, Gloor GB, Edgell DR. Nucleic Acids Research. (2010)
     

80. Strand-specific contacts and divalent metal ion regulate double-strand break formation by the GIY-YIG homing endonuclease I-BmoI.
    Carter JM, Friedrich NC, Kleinstiver B, Edgell DR. Journal of Molecular Biology. (2007)