SCIENTIFIC POSTERS & PUBLICATIONS
SCIENTIFIC POSTERS & PUBLICATIONS

May 18, 2023
American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting
Small nuclear RNA-Mediated Exon 51 Skipping AAV9 Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
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May 18, 2023
American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting
Cas13d Multi-Targeting Efficiently Targets Sense and Antisense HRE Containing Toxic RNAs and Poly-GP DPR in C9ALS Patient Cells and in C9-BAC500 Mouse Model
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May, 2023
Strategy for AAV packaging of multiple snRNAs with genome integrity
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Poster: AAV9 Mediated Delivery of RNA Targeting Systems Corrects Molecular and Functional Defects in Myotonic Dystrophy Type 1
November 18, 2021
Society for Muscle Biology: Frontiers in Myogenesis Conference
AAV9 Mediated Delivery of RNA Targeting Systems Corrects Molecular and Functional Defects in Myotonic Dystrophy Type 1
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Poster: RNA-Targeting Gene Therapy Exon-Skipping Strategy to Treat Genetic Diseases
May 31, 2021
26th Annual Meeting of the RNA Society
RNA-Targeting Gene Therapy Exon-Skipping Strategy to Treat Genetic Diseases
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Presentation: AAV9 Mediated Delivery of PUF RNA Targeting System Corrects Molecular and Functional Defects in a Myotonic Dystrophy Type 1 Mouse Model
May 13, 2021
American Society for Gene and Cell Therapy 24th Annual Meeting
AAV9 Mediated Delivery of PUF RNA Targeting System Corrects Molecular and Functional Defects in a Myotonic Dystrophy Type 1 Mouse Model (Oral Presentation)
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Poster: Novel RNA-Targeting Gene Therapy Approach for Usher’s Syndrome Type II Retinitis Pigmentosa
May 11, 2021
American Society for Gene and Cell Therapy 24th Annual Meeting
Novel RNA-Targeting Gene Therapy Approach for Usher’s Syndrome Type II Retinitis Pigmentosa (Poster ID #100)
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Poster: Rational Design of Short Constitutive Promoters for Improved Transgene Expression in AAV Vectors
May 11, 2021
American Society for Gene and Cell Therapy 24th Annual Meeting
Rational Design of Short Constitutive Promoters for Improved Transgene Expression in AAV Vectors (Poster ID #281)
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Publication: The Sustained Expression of Cas9 Targeting Toxic RNAs Reverses Disease Phenotypes in Mouse Models of Myotonic Dystrophy Type 1
September 14, 2020
nature biomedical engineering
The Sustained Expression of Cas9 Targeting Toxic RNAs Reverses Disease Phenotypes in Mouse Models of Myotonic Dystrophy Type 1
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Poster: RNA-targeting Gene Therapy for Human Genetic Diseases
May 27, 2020
Locana Participates in the 25th Annual Meeting of the RNA Society to Share Research Regarding RNA-targeting Gene Therapy for Human Genetic Diseases
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Poster: AAV9 Mediated Delivery of RNA Targeting CRISPR Corrects Molecular and Functional Phenotypes in Myotonic Dystrophy Type 1 Mouse Model
May 13, 2020
Locana Presents Poster at ASGCT 2020: AAV9 Mediated Delivery of RNA Targeting CRISPR Corrects Molecular and Functional Phenotypes in Myotonic Dystrophy Type 1 Mouse Model
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Publication: Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9
August 24, 2017
CELL
Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9
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