CA3017956A1 - Crispr/cas-related methods and compositions for treating beta hemoglobinopathies - Google Patents

Crispr/cas-related methods and compositions for treating beta hemoglobinopathies Download PDF

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Publication number
CA3017956A1
CA3017956A1 CA3017956A CA3017956A CA3017956A1 CA 3017956 A1 CA3017956 A1 CA 3017956A1 CA 3017956 A CA3017956 A CA 3017956A CA 3017956 A CA3017956 A CA 3017956A CA 3017956 A1 CA3017956 A1 CA 3017956A1
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nucleic acid
nucleotides
domain
molecule
hbg1
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CA3017956A
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French (fr)
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Jennifer Leah GORI
Luis A. BARRERA
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Editas Medicine Inc
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Editas Medicine Inc
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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CA3017956A 2016-03-14 2017-03-14 Crispr/cas-related methods and compositions for treating beta hemoglobinopathies Pending CA3017956A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662308190P 2016-03-14 2016-03-14
US62/308,190 2016-03-14
US201762456615P 2017-02-08 2017-02-08
US62/456,615 2017-02-08
PCT/US2017/022377 WO2017160890A1 (en) 2016-03-14 2017-03-14 Crispr/cas-related methods and compositions for treating beta hemoglobinopathies

Publications (1)

Publication Number Publication Date
CA3017956A1 true CA3017956A1 (en) 2017-09-21

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CA3017956A Pending CA3017956A1 (en) 2016-03-14 2017-03-14 Crispr/cas-related methods and compositions for treating beta hemoglobinopathies

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US (2) US20200255857A1 (enExample)
EP (1) EP3430142A1 (enExample)
JP (3) JP2019508051A (enExample)
KR (2) KR102532663B1 (enExample)
CN (4) CN118127006A (enExample)
AU (3) AU2017235333B2 (enExample)
CA (1) CA3017956A1 (enExample)
IL (2) IL313038A (enExample)
MX (2) MX2018011114A (enExample)
SG (1) SG11201807859WA (enExample)
WO (1) WO2017160890A1 (enExample)

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