Pablo Perez-Pinera

Research Interests

• Neuromuscular diseases
• Neurodegenerative diseases
• Biopharmaceutical Production
• Gene Therapy
• Regenerative Medicine
• Genome Engineering
• Synthetic Biology

Primary Research Area

• Synthetic Bioengineering

Research Areas

• Biomolecular engineering
• Gene and cell therapy
• Genome engineering
• Regenerative medicine
• Regenerative tissue engineering

Selected Articles in Journals

• Gaj T, Perez-Pinera P. Next Generation Genome Editing. Curr Opin Biomed Eng. 2024
• Gapinske M, Winter J, Swami D, Gapinske L, Woods WS, Shirguppe S, Miskalis A, Busza A, Joulani D, Kao CJ, Kostan K, Bigot A, Bashir R, Perez-Pinera P. Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors. Mol Ther Nucleic Acids. 2023 Jul 27;33:572-586.
• Lim CKW, McCallister TX, Saporito-Magriña C, McPheron GD, Krishnan R, Zeballos C MA, Powell JE, Clark LV, Perez-Pinera P*, Gaj T*. CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genes. Mol Ther. 2022 Dec 7;30(12):3619-3631. *Co-corresponding authors.
• Lim CKW, Gapinske M, Brooks AK, Woods WS, Powell JE, Zeballos C MA, Winter J, Perez-Pinera P*, Gaj T*. Treatment of a Mouse Model of ALS by In Vivo Base Editing. Mol Ther. 2020 Apr 8;28(4):1177-1189. *Co-corresponding authors.
• Winter J, Luu A, Gapinske M, Manandhar S, Shirguppe S, Woods WS, Song JS, Perez-Pinera P. Targeted Exon Skipping with AAV-Mediated Split Adenine Base Editors. Cell Discovery. 2019 Aug 20;5:41
• Brown A, Winter J, Gapinske M, Tague N, Woods WS, Perez-Pinera P. Multiplexed and tunable transcriptional activation by promoter insertion using nuclease-assisted vector integration. Nucleic Acids Res. 2019 Jul 9;47(12):e67
• CRISPR-SKIP: programmable gene splicing with single base editors. Gapinske M, Luu A, Winter J, Woods WS, Kostan KA, Shiva N, Song JS, Perez-Pinera P. Genome Biol. 2018 Aug 15;19(1):107.
• Synergistic and tunable human gene activation by combinations of synthetic transcription factors. Perez-Pinera P, Ousterout DG, Brunger JM, Farin AM, Glass KA, Guilak F, Crawford GE, Hartemink AJ, Gersbach CA. Nat Methods. 2013 Mar;10(3):239-42.
• RNA-guided gene activation by CRISPR-Cas9-based transcription factors. Perez-Pinera P, Kocak DD, Vockley CM, Adler AF, Kabadi AM, Polstein LR, Thakore PI, Glass KA, Ousterout DG, Leong KW, Guilak F, Crawford GE, Reddy TE, Gersbach CA. Nat Methods. 2013 Oct;10(10):973-6.

Pending Articles

• 1. Miskalis A, Shirguppe S, Winter J, Elias G, Swami D, Nambiar A, Stilger M, Woods W, Gosstola N, Gapinske M, Zeballos A, Moore H, Maslov S, Gaj T and Perez-Pinera P. SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping. Under review Nat Commun.