Research Topics

• Synthetic Bioengineering

For more information

• Genome Engineering and Transcriptional Regulation Laboratory

Research Topics

• Synthetic Bioengineering

Research Interests

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

Selected Articles in Journals

• 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.
• Multiplexed Targeted Genome Engineering Using a Universal Nuclease-Assisted Vector Integration System. Brown A, Woods WS, Perez-Pinera P. ACS Synth Biol. 2016 Jul 15;5(7):582-8.
• Synthetic biology and microbioreactor platforms for programmable production of biologics at the point-of-care. Perez-Pinera P, Han N, Cleto S, Cao J, Purcell O, Shah KA, Lee K, Ram R, Lu TK. Nat Commun. 2016 Jul 29;7:12211.
• Biomedical applications of gene editing. Perez-Pinera P*, Chen ZY. Hum Genet. 2016 Sep;135(9):967-9. *Corresponding author.
• 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.
• Disruption of the β1L Isoform of GABP Reverses Glioblastoma Replicative Immortality in a TERT Promoter Mutation-Dependent Manner. Mancini A, Xavier-Magalhães A, Woods WS, Nguyen KT, Amen AM, Hayes JL, Fellmann C, Gapinske M, McKinney AM, Hong C, Jones LE, Walsh KM, Bell RJA, Doudna JA, Costa BM, Song JS, Perez-Pinera P, Costello JF. Cancer Cell. 2018 Sep 10;34(3):513-528
• 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
• 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. Aug. 20.