Infectious Diseases – Jasdeep MANN, Joel Gay, Jordan JARJOUR, Joy Zhang, Bluebird Bio Inc, 2Seventy Bio Inc
Abstract for “Pd-1 homing Endonuclease Variants, Compositions and Methods of Use”
“The present disclosure provides improved genome editing compositions, and methods for editing a PD-1 genes. Genome edited cells are also provided in the disclosure for the treatment or prevention of at least one symptom, such as cancer, infectious disease, autoimmune disease, inflammation disease or immunodeficiency.
Background for “Pd-1 homing Endonuclease Variants, Compositions and Methods of Use”
“Technical Field”
“Description of Related Art”
“BRIEF DESCRIPTION ABOUT CERTAIN VIEWS OF THE DRAWINGS.”
“BRIEF DESCRIPTION ABOUT THE SEQUENCE IDENTIFIERS.”
“A. Overview”
“B. Definitions”
“C. Nuclease Variants.”
“D. Target Sites.”
“E. Donor Repair Templates”
“F. Polypeptides”
“G. Polynucleotides”
“H. Genome Edited cells”
“I. Formulations and Compositions”
“J. Genome Edited cellular Therapies”
“EXAMPLES”
“Example 1”
“Reprogramming IonuI to Disrupt Intracellular Signaling Motif in the Programmed Death Receptor-1 Gene (Pd-1)”
“Example 2”
“Characterization an I-OnuI Variant, and a MegaTAL Targeting PD-1 Exon 5”
“Example 3”
“Reprogramming IonuI Meganuclease domains to Disrupt Extracellular Domains in the Programmed Death Receptor-1 Gene (Pd-1)”
“Example 4”
“Targeted Disruption in Primary Human T Cells of the Pd-1 Gene Rescues T Cell Function Suppression by Pd-L1”
“Example 5”
“Homologous recombination of a Transgene to Exon 1 of Pd-1 Gene”
“Example 6”
“Homologous Recombination a Transgene Encoding a Chmeric Antigen Receptor(CAR) into PD-1 Gene
“Example 7”
“Homologous Recombination a Promoter-Less transgene into PD-1 gene”
“Example 8”
“Constitutive PD-1 expression following homologous recombination of a transgene encoding a fluorescent protein into Exon 5 PD-1 gene”
“Example 9”
“Homologous recombination of a Transgene to the PD-1 Gene Is Independent of Single Nucleotide Polymorphisms (SNPS).”
“Example 10”
“Homologous recombination of a Transgene Coding a Switch Receiver into the PDCD1 locus”
“Example 11”
“Long-Range Excision PD-1 Intracellular Signaling Area Using HDR”
“Example 12”
“PD-1 Gene Disruption in Primary Human T Cells In Vitro and Antibody-Mediated PD1 Blocked”
“Example 13”
“Homologous Recombination a Promoter-Less transgene into PD-1 gene”
“Example 14”
“Homologous Recombination a Promoter-Less Cytokine Transgene Into the PD-1 Locus
Summary for “Pd-1 homing Endonuclease Variants, Compositions and Methods of Use”
“Technical Field”
“Description of Related Art”
“BRIEF DESCRIPTION ABOUT CERTAIN VIEWS OF THE DRAWINGS.”
“BRIEF DESCRIPTION ABOUT THE SEQUENCE IDENTIFIERS.”
“A. Overview”
“B. Definitions”
“C. Nuclease Variants.”
“D. Target Sites.”
“E. Donor Repair Templates”
“F. Polypeptides”
“G. Polynucleotides”
“H. Genome Edited cells”
“I. Formulations and Compositions”
“J. Genome Edited cellular Therapies”
“EXAMPLES”
“Example 1”
“Reprogramming IonuI to Disrupt Intracellular Signaling Motif in the Programmed Death Receptor-1 Gene (Pd-1)”
“Example 2”
“Characterization an I-OnuI Variant, and a MegaTAL Targeting PD-1 Exon 5”
“Example 3”
“Reprogramming IonuI Meganuclease domains to Disrupt Extracellular Domains in the Programmed Death Receptor-1 Gene (Pd-1)”
“Example 4”
“Targeted Disruption in Primary Human T Cells of the Pd-1 Gene Rescues T Cell Function Suppression by Pd-L1”
“Example 5”
“Homologous recombination of a Transgene to Exon 1 of Pd-1 Gene”
“Example 6”
“Homologous Recombination a Transgene Encoding a Chmeric Antigen Receptor(CAR) into PD-1 Gene
“Example 7”
“Homologous Recombination a Promoter-Less transgene into PD-1 gene”
“Example 8”
“Constitutive PD-1 expression following homologous recombination of a transgene encoding a fluorescent protein into Exon 5 PD-1 gene”
“Example 9”
“Homologous recombination of a Transgene to the PD-1 Gene Is Independent of Single Nucleotide Polymorphisms (SNPS).”
“Example 10”
“Homologous recombination of a Transgene Coding a Switch Receiver into the PDCD1 locus”
“Example 11”
“Long-Range Excision PD-1 Intracellular Signaling Area Using HDR”
“Example 12”
“PD-1 Gene Disruption in Primary Human T Cells In Vitro and Antibody-Mediated PD1 Blocked”
“Example 13”
“Homologous Recombination a Promoter-Less transgene into PD-1 gene”
“Example 14”
“Homologous Recombination a Promoter-Less Cytokine Transgene Into the PD-1 Locus
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