TREATMENT OF KRABBE DISEASE WITH UMBILICAL CORD BLOOD TRANSPLANTION (UCBT) AND INCREASED GALACTOCEREBROSIDASE (GALC) EXPRESSION

• Main Authors: SZABOLCS PAUL, ESCOLAR MARIA LUISA
• Format: Patent
• Language: Chinese; English
• Published: 23.08.2019
• 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; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

This application provides methods of treating Krabbe disease, for example in an infant. Such methods can include immunosuppressing the patient, for example by administration of a myeloablative regimen, administering an umbilical cord blood transplant (UCBT) (such as an allogenic UCBT), and increasing expression of galactocerebrosidase (GALC) in the patient (e.g., by using gene editing). 本申请提供治疗克拉伯病的方法，例如治疗婴儿中克拉伯病的方法。这样的方法包括免疫抑制患者(例如通过施用清髓方案)，施用脐带血移植(UCBT)(如同种异体的UCBT)，和增加患者中半乳糖脑苷脂酶(GALC)的表达(例如，通过使用基因编辑)。