Technologies enabling the next generation of gene therapy, including nucleic acid editing and delivery systems. Gene Editing S22-282 Enhanced mammalian CRISPR editing with separated retron donor and nickases Lars Steinmetz Kevin Roy Dominik Lindenhofer S22-089 Viral gene transfer of mutant CRISPR/Cas9 to modulate human alpha-synuclein expression as a precision gene therapy for Parkinson's disease Carmen Alejandra Torres Lei Qi Deniz Kirik Birgitt Schuele Faria Zafar S21-085 Using high-throughput single-strand DNA profiling for profiling CRISPR targeting of DNA sequences (CasKAS) Georgi Marinov Chuan He William Greenleaf Tong Wu S21-197 Correction of polycythemia vera via CRISPR/AAV6 genome editing Matthew Porteus Michael Cromer S21-023 CRISPR-based FOXP3 gene Engineered T cells and Hematopoietic Stem Cell Precursors to treat IPEX syndrome patients Rosa Bacchetta Maria-Grazia Roncarolo Matthew Porteus Marianne Goodwin S21-175 Potential Curative Treatment for Alpha-Thalassemia Using CRISPR-Mediated Genome Editing Matthew Porteus Michael Cromer Tippi MacKenzie Beeke Weinert S21-459 CRISPR-based gene editing platform to treat FOXP3-mediated immune dysregulation Rosa Bacchetta Maria-Grazia Roncarolo Matthew Porteus Esmond Lee Simon Borna S20-412 Gene Editing and Delivery of Myeloid cells To Promote Wound Healing Geoffrey Gurtner Dominic Henn Lei Qi Dehua Zhao S20-282 Super Recombinator (SuRe), a CRISPR/Cas9-based platform for the rapid incorporation of multiple transgenes at the same genetic locus Mark Schnitzer Cheng Huang Junjie Luo S20-412 Gene Editing and Delivery of Myeloid cells To Promote Wound Healing Geoffrey Gurtner Dominic Henn Lei Qi Dehua Zhao S20-082 Antiviral CRISPR Systems for Modulating Host Immune Response and Targeting the Virus Genome Lei Qi Marie La Russa Timothy Abbott David Lewis S20-414 High-Multiplexed Genome Engineering Using Synthetic CRISPR Arrays Lei Qi Jens Magnusson S19-079 GOTI- method to identify off target mutations caused by gene editors Hui Yang Yixue Li Lars Steinmetz Erwei Zuo Yidi Sun Wu Wei S18-496 Control of gene editing and gene expression via integrated anti-CRISPR proteins Lei Qi Muneaki Nakamura S18-365 CRISPR-GO: A Method for Programmable Genome Re-organization Lei Qi Haifeng Wang S16-369 CLOuD9- a method for CRISPR-mediated chromosomal looping Kevin Wang Stefanie Morgan Fangting Wu Chiao-Chain Huang S15-441 CRISPR/Cas9-mediated Genome Editing to Treat EGFR-mutant Lung Cancer Huibin Tang Joseph Shrager S17-475 Programmed CRISPR-Cas nucleases are potent RNA-guided nickases Xu Hua Fu Andrew Fire Justin Smith S16-009 Genome Editing of Human Neuronal Stem Cells (NSCs) to Treat Genetic Diseases of the CNS, Neural Injury, and Neurodegenerative Diseases Matthew Porteus Eric Kildebeck Daniel Dever Joseph Clark S16-025 High-throughput precision genome editing Zachery Smith Hunter Fraser Gene Delivery S17-343 Exosome platform for tissue-specific drug delivery of mRNA or other therapeutic cargo Jing-Hung Wang Alexis Forterre A.C. Matin Alain Delcayre S16-157 CART- a transfection delivery system for efficient intracellular mRNA delivery Robert Waymouth Paul Wender Jessica Vargas Timothy Blake Colin McKinlay Ronald Levy Nancy Benner Katherine Keenan Ole Audun Haabeth S22-152 AAV-LK03 Variant With Enhanced Transduction Properties in Humans and Rodents Mark Kay Adriana Gonzalez Sandoval S11-298 Chimeric AAV Capsids for High Efficiency Nucleic Acid Transfer Leszek Lisowski Mark Kay S15-240 Controlling AAV receptor expression to improve testing and validation of AAV gene therapy products Sirika Wood Jan Carette Michael Chapman Nancy Meyer Andreas Puschnik Omar Davulcu S16-453 Efficient homologous recombination of large transgenes using AAV donor vectors Matthew Porteus Rasmus Bak Sriram Vaidyanathan S18-520 Enhancing AAV-mediated gene targeting with ribonucleotide reductase inhibitors Mark Kay Shinnosuke Tsuji S00-013 Increasing the size of rAAV-mediated expression cassettes in vivo Mark Kay Hiroyuki Nakai S15-305 Novel AAV Capsids Resistant to Pre-existing Human Neutralizing Antibodies Nicole Paulk Mark Kay S06-098 Novel AAV capsids with new transduction and nonimmune properties Mark Kay Dirk Grimm S17-319 Pancreatic islet cell-specific rAAV vectors for more efficient gene therapy or research Katja Pekrun Mark Kay S15-007 Method to improve effectiveness of gene therapy for Duchenne muscular dystrophy Lawrence Steinman Peggy Ho
S22-282 Enhanced mammalian CRISPR editing with separated retron donor and nickases Lars Steinmetz Kevin Roy Dominik Lindenhofer
S22-089 Viral gene transfer of mutant CRISPR/Cas9 to modulate human alpha-synuclein expression as a precision gene therapy for Parkinson's disease Carmen Alejandra Torres Lei Qi Deniz Kirik Birgitt Schuele Faria Zafar
S21-085 Using high-throughput single-strand DNA profiling for profiling CRISPR targeting of DNA sequences (CasKAS) Georgi Marinov Chuan He William Greenleaf Tong Wu
S21-197 Correction of polycythemia vera via CRISPR/AAV6 genome editing Matthew Porteus Michael Cromer
S21-023 CRISPR-based FOXP3 gene Engineered T cells and Hematopoietic Stem Cell Precursors to treat IPEX syndrome patients Rosa Bacchetta Maria-Grazia Roncarolo Matthew Porteus Marianne Goodwin
S21-175 Potential Curative Treatment for Alpha-Thalassemia Using CRISPR-Mediated Genome Editing Matthew Porteus Michael Cromer Tippi MacKenzie Beeke Weinert
S21-459 CRISPR-based gene editing platform to treat FOXP3-mediated immune dysregulation Rosa Bacchetta Maria-Grazia Roncarolo Matthew Porteus Esmond Lee Simon Borna
S20-412 Gene Editing and Delivery of Myeloid cells To Promote Wound Healing Geoffrey Gurtner Dominic Henn Lei Qi Dehua Zhao
S20-282 Super Recombinator (SuRe), a CRISPR/Cas9-based platform for the rapid incorporation of multiple transgenes at the same genetic locus Mark Schnitzer Cheng Huang Junjie Luo
S20-412 Gene Editing and Delivery of Myeloid cells To Promote Wound Healing Geoffrey Gurtner Dominic Henn Lei Qi Dehua Zhao
S20-082 Antiviral CRISPR Systems for Modulating Host Immune Response and Targeting the Virus Genome Lei Qi Marie La Russa Timothy Abbott David Lewis
S19-079 GOTI- method to identify off target mutations caused by gene editors Hui Yang Yixue Li Lars Steinmetz Erwei Zuo Yidi Sun Wu Wei
S18-496 Control of gene editing and gene expression via integrated anti-CRISPR proteins Lei Qi Muneaki Nakamura
S16-369 CLOuD9- a method for CRISPR-mediated chromosomal looping Kevin Wang Stefanie Morgan Fangting Wu Chiao-Chain Huang
S15-441 CRISPR/Cas9-mediated Genome Editing to Treat EGFR-mutant Lung Cancer Huibin Tang Joseph Shrager
S17-475 Programmed CRISPR-Cas nucleases are potent RNA-guided nickases Xu Hua Fu Andrew Fire Justin Smith
S16-009 Genome Editing of Human Neuronal Stem Cells (NSCs) to Treat Genetic Diseases of the CNS, Neural Injury, and Neurodegenerative Diseases Matthew Porteus Eric Kildebeck Daniel Dever Joseph Clark
S17-343 Exosome platform for tissue-specific drug delivery of mRNA or other therapeutic cargo Jing-Hung Wang Alexis Forterre A.C. Matin Alain Delcayre
S16-157 CART- a transfection delivery system for efficient intracellular mRNA delivery Robert Waymouth Paul Wender Jessica Vargas Timothy Blake Colin McKinlay Ronald Levy Nancy Benner Katherine Keenan Ole Audun Haabeth
S22-152 AAV-LK03 Variant With Enhanced Transduction Properties in Humans and Rodents Mark Kay Adriana Gonzalez Sandoval
S15-240 Controlling AAV receptor expression to improve testing and validation of AAV gene therapy products Sirika Wood Jan Carette Michael Chapman Nancy Meyer Andreas Puschnik Omar Davulcu
S16-453 Efficient homologous recombination of large transgenes using AAV donor vectors Matthew Porteus Rasmus Bak Sriram Vaidyanathan
S18-520 Enhancing AAV-mediated gene targeting with ribonucleotide reductase inhibitors Mark Kay Shinnosuke Tsuji
S15-305 Novel AAV Capsids Resistant to Pre-existing Human Neutralizing Antibodies Nicole Paulk Mark Kay
S17-319 Pancreatic islet cell-specific rAAV vectors for more efficient gene therapy or research Katja Pekrun Mark Kay
S15-007 Method to improve effectiveness of gene therapy for Duchenne muscular dystrophy Lawrence Steinman Peggy Ho