Engineering self-deliverable ribonucleoproteins for genome editing in the brain

Chen K, Stahl EC, Kang MH, Xu B, Allen R, Trinidad M, and Doudna JA. Nature Communications

(2024)

Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection

Tuma J, Chen YJ, Collins MG, Paul A, Li J, Han H, Sharma R, Murthy N, Lee HY. Biochemistry

(2023)

Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs

Stahl EC, Sabo JK, Kang MH, Allen R, Applegate E, Kim SE, Kwon Y, Seth A, Lemus N, Salinas-Rios V, Soczek KM, Trinidad M, Vo LT, Jeans C, Wozniak A, Morris T, Kimberlin A, Foti T, Savage DF, and Doudna JA. Molecular Therapy

(2023)

Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes

Foss DV, Muldoon JJ, Nguyen DN, Carr D, Sahu SU, Hunsinger JM, Wyman SK, Krishnappa N, Mendonsa R, Schanzer EV, Shy BR, Vykunta VS, Allain C, Li Z, Marson A, Eyquem J, and Wilson RC. Nature Biomedical Engineering

(2023)

Outsmarting delivery barriers in vivo: Base editing via next-generation virus-like particles

Wilson RC. GEN Biotechnology 

(2022)

Acid-sensitive surfactants enhance the delivery of nucleic acids

Røise JJ, Han H, Li J, Kerr DL, Taing C, Behrouzi K, He M, Ruan E, Chan LY, Espinoza EM, Reinhard S, Thakker K, Kwon J, Mofrad MRK, and Murthy N. Molecular Pharmaceutics

(2021)

The coiled-coil forming peptide (KVSALKE)5 is a cell penetrating peptide that enhances the intracellular delivery of proteins

Li J, Tuma J, Han H, Kim H, Wilson RC, Lee HY, and Murthy N. Advanced Healthcare Materials

(2021)

Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering

Hamilton JR, Tsuchida CA, Nguyen DN, Shy BR, McGarrigle ER, Espinoza CRS, Carr D, Blaeschke F, Marson A, and Doudna JA. Cell Reports

(2021)

Site-specific bioconjugation through enzyme-catalyzed tyrosine-cysteine bond formation

Lobba MJ, Fellmann C, Marmelstein AM, Maza JC, Kissman EN, Robinson SA, Staahl BT, Urnes C, Lew RJ, Mogilevsky CS, Doudna JA, Francis MB. ACS Central Science

(2020)

Engineering of monosized lipid-coated mesoporous silica nanoparticles for CRISPR delivery

Noureddine A, Maestas-Olguin A, Saada EA, LaBauve AE, Agola JO, Baty KE, Howard T, Sabo JK, Espinoza CRS, Doudna JA, Schoeniger JS, Butler KS, Negrete OA, Brinker CJ, Serda RE. Acta Biomaterialia

(2020)

Non-viral strategies for delivering genome editing enzymes

Li J, Justad Røise J, He M, Das R, Murthy N. Advanced Drug Delivery Reviews

(2020)

A traceless linker for aliphatic amines that rapidly and quantitatively fragments after reduction.

He M, Li J, Han H, Borges CA, Neiman G, Røise JJ, Hadaczek P, Mendonsa R, Holm VR, Wilson RC, Bankiewicz K, Zhang Y, Sadlowski CM, Healy K, Riley LW, and Murthy N. Chemical Science

(2020)

The delivery challenge: fulfilling the promise of therapeutic genome editing.

Haasteren JV, Li J, Scheideler OJ, Murthy N, and Schaffer DV. Nature Biotechnology

(2020)

Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency.

Nguyen DN, Roth TL, Li PJ, Chen PA, Apathy R, Mamedov MR, Vo LT, Tobin VR, Goodman D, Shifrut E, Bluestone JA, Puck JM, Szoka FC, and Marson A. Nature Biotechnology

(2019)

The daunting economics of therapeutic genome editing.

Wilson RC and Carroll D. The CRISPR Journal

(2019)

Nontoxic nanopore electroporation for effective intracellular delivery of biological macromolecules

Cao Y, Ma E, Cestellos-Blanco S, Zhang B, Qiu R, Su Y, Doudna JA, Yang P. Proceedings of the National Academy of Sciences

(2019)

A novel fluorescent surfactant enhances the delivery of the Cas9 ribonucleoprotein and enables the identification of edited cells

Li J, Røise JJ, Zhang J, Yang J, Kerr DL, Han H, Murthy N. Chemical Communications 

(2019)

CRISPR-Cas9 genome engineering of primary CD4+ T cells for the interrogation of HIV-host factor interactions

Hultquist JF, Hiatt J, Schumann K, McGregor MJ, Roth TL, Haas P, Doudna JA, Marson A, Krogan NJ. Nature Protocols

(2019)

Engineering CRISPR-Cas9 RNA–protein complexes for improved function and delivery.

Rouet R, de Oñate L, Li J, Murthy N, and Wilson RC. The CRISPR Journal

(2018)

Emerging strategies for genome editing in the brain.

Foss DV and Wilson RC. Trends in Molecular Medicine (2018)

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

Lee B, Lee K, Panda S, Gonzales-Rojas R, Chong A, Bugay V, Park HM, Brenner R, Murthy N, Lee HY. Nature Biomedical Engineering

(2018)

Genetic engineering in primary human B cells with CRISPR-Cas9 ribonucleoproteins

Wu CM, Roth TL, Baglaenko Y, Ferri DM, Brauer P, Zuniga-Pflucker JC, Rosbe KW, Wither JE, Marson A, Allen CDC. Genetic engineering in primary human B cells with CRISPR-Cas9 ribonucleoproteins. Journal of Immunological Methods

(2018)

Enhanced genome editing with Cas9 ribonucleoprotein in diverse cells and organisms.

Farboud B, Jarvis E, Roth TL, Shin J, Corn JE, Marson A, Meyer BJ, Patel NH, and Hochstrasser ML. Journal of Visualized Experiments

(2018)

Receptor-mediated delivery of CRISPR-Cas9 endonuclease for cell type specific gene editing.

Rouet R, Thuma BA, Roy MD, Lintner NG, Rubitski DM, Finley JE, Wisniewska HM, Mendonsa R, Hirsh A, de Oñate L, Compte J, Mclellan TJ, Bellenger J, Feng X, Varghese A, Chrunyk BA, Borzilleri KA, Hesp KD, Zhou K, Ma N, Tu M, Dullea R, Mcclure KF, Wilson RC, Liras S, Mascitti V, and Doudna JA. JACS (2018)

The promise and challenge of in vivo delivery for genome therapeutics.

Wilson RC and Gilbert LA. ACS Chemical Biology

(2017)

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

Lee K, Conboy M, Park HM, Jiang F, Kim HJ, Dewitt MA, Mackley VA, Chang K, Rao A, Skinner C, Shobha T, Mehdipour M, Liu H, Huang WC, Lan F, Bray NL, Li S, Corn JE, Kataoka K, Doudna JA, Conboy I, Murthy N. Nature Biomedical Engineering

(2017)

Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery

Gaj T, Staahl BT, Rodrigues GMC, Limsirichai P, Ekman FK, Doudna JA, Schaffer DV. Nucleic Acids Research

(2017)

Synthetically modified guide RNA and donor DNA are a versatile platform for CRISPR-Cas9 engineering.

Lee K, Mackley VA, Rao A, Chong AT, Dewitt MA, Corn JE, and Murthy N. eLife (2017)

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes

Staahl BT, Benekareddy M, Coulon-Bainier C, Banfal AA, Floor SN, Sabo JK, Urnes C, Munares GA, Ghosh A, Doudna JA. Nature Biotechnology

(2017)

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

Schumann K, Lin S, Boyer E, Simeonov DR, Subramaniam M, Gate RE, Haliburton GE, Ye CJ, Bluestone JA, Doudna JA, Marson A. Generation of knock-in primary human T cells using Cas9 ribonucleoproteins. Proceedings of the National Academy of Sciences

(2015)

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

Lin S, Staahl BT, Alla RK, Doudna JA. eLife

(2014)