IGI CRISPR Delivery Collective
IGI CRISPR Delivery Collective
Developing new technology for delivery of CRISPR-based therapies
Research
The IGI CRISPR Delivery Collective is a collaborative effort driven by five labs — Doudna, Marson, Murthy, Nguyen and Wilson — working to develop new technology that enables or improves access to therapeutic genome editing.
The CRISPR delivery challenge is the single greatest impediment to widespread use of CRISPR-based therapies for genetic diseases and beyond. There is an urgent need for new approaches for getting genome-editing enzymes into therapeutically-relevant cells, and we are using every trick in the book to enable safe, effective, and accessible clinical genome editing. Although viral vectors dominate the pre-clinical landscape, we believe that non-viral CRISPR delivery holds great untapped potential.
By bringing together scientists from diverse backgrounds, we will advance creative strategies for therapeutic editing of immune cells, the lungs, blood-generating stem cells, the brain, and beyond.
People
CRISPR Delivery Publications
Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) in primary human immune cells and hematopoietic stem cells
Sahu SU, Castro M, Muldoon JJ, Asija K, Wyman SK, Krishnappa N, de Oñate L, Eyquem J, Nguyen DN, and Ross C. Wilson. Nature Protocols
Packaged delivery of CRISPR–Cas9 ribonucleoproteins accelerates genome editing
Karp H, Zoltek M, Wasko K, Vazquez AL, Brim J, Ngo W, Schepartz A, and Jennifer A Doudna. Nucleic Acids Research
Mechanism-guided engineering of a minimal biological particle for genome editing
Ngo W, Peukes J, Baldwin A, Xue ZW, Hwang S, Stickels RR, Lin Z, Satpathy AT, Wells JA, Schekman R, Nogales E, and Doudna JA. PNAS
Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein
Chen K, Han H, Zhao S, Xu B, Yin B, Lawanprasert A, Trinidad M, Burgstone BW, Murthy N, Doudna JA. Nature Biotechnology
Ethylene oxide graft copolymers reduce the immunogenicity of lipid nanoparticles
Qi Y, Han H, Liu A, Zhao S, Lawanprasert A, Nielsen JE, Choudhary H, Liang D, Barron AE, and Murthy N. RSC Advances
Acid-degradable lipid nanoparticles enhance the delivery of mRNA
Zhao S, Gao K, Han H, Stenzel M, Yin B, Song H, Lawanprasert A, Nielsen JE, Sharma R, Arogundade OH, Pimcharoen S, Chen Y-J, Paul A, Tuma J, Collins MG, Wyle Y, Cranick MG, Burgstone BW, Perez BS, Barron AE, Smith AM, Lee HY, Wang A, and Murthy N. Nature Nanotechnology
Targeted nonviral delivery of genome editors in vivo
Tsuchida CA, Wasko KM, Hamilton JR, and Doudna JA. PNAS
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
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
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
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
Outsmarting delivery barriers in vivo: Base editing via next-generation virus-like particles
Wilson RC. GEN Biotechnology
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
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
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
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
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
Non-viral strategies for delivering genome editing enzymes
Li J, Justad Røise J, He M, Das R, Murthy N. Advanced Drug Delivery Reviews
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
The delivery challenge: fulfilling the promise of therapeutic genome editing.
Haasteren JV, Li J, Scheideler OJ, Murthy N, and Schaffer DV. Nature Biotechnology
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
The daunting economics of therapeutic genome editing.
Wilson RC and Carroll D. The CRISPR Journal
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
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
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
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
Emerging strategies for genome editing in the brain.
Foss DV and Wilson RC. Trends in Molecular Medicine
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
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
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
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
The promise and challenge of in vivo delivery for genome therapeutics.
Wilson RC and Gilbert LA. ACS Chemical Biology
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
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
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
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
Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery
Lin S, Staahl BT, Alla RK, Doudna JA. eLife
Contact
For any inquiries, get in touch with Ross Wilson, IGI Delivery Collective coordinator: rosswilson@berkeley.edu or +1-510-542-9352
Learn More
Read more about therapeutic CRISPR delivery in CRISPRpedia: