MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES

Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

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Hauptverfasser: Chakraborty, Tirtha, Cowan, Chad Albert, Kernytsky, Andrew, Borland, Todd Douglass, Paik, Elizabeth Jae-eun, Lundberg, Ante Sven, Lin, Michelle l-ching, Mishra, Bibhu Prasad
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creator Chakraborty, Tirtha
Cowan, Chad Albert
Kernytsky, Andrew
Borland, Todd Douglass
Paik, Elizabeth Jae-eun
Lundberg, Ante Sven
Lin, Michelle l-ching
Mishra, Bibhu Prasad
description Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
title MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
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