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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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. |
format | Patent |
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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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