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1. (WO2015117027) AN IMPROVED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2015/117027 International Application No.: PCT/US2015/013960
Publication Date: 06.08.2015 International Filing Date: 30.01.2015
IPC:
A61K 48/00 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants:
CHILDREN'S HOSPITAL MEDICAL CENTER [US/US]; 3333 Burnet Avenue, ML7032 Cincinnati, Ohio 45229-3039, US
Inventors:
MALIK, Punam; US
Agent:
CHEN, Yahua; US
Priority Data:
61/933,78830.01.2014US
Title (EN) AN IMPROVED FETAL HEMOGLOBIN FOR GENETIC CORRECTION OF SICKLE CELL DISEASE
(FR) HÉMOGLOBINE FŒTALE AMÉLIORÉE POUR LA CORRECTION GÉNÉTIQUE DE LA DRÉPANOCYTOSE
Abstract:
(EN) Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or β-thalassemia.
(FR) La présente invention concerne des méthodes et des compositions se rapportant d'une manière générale à des méthodes de détermination du chimérisme minimum de cellules souches hématopoïétiques (HSC) et un dosage génique pour la correction d'une maladie hématopoïétique ; en particulier, dans des modèles in vivo. L'invention concerne également des vecteurs d'expression lentiviraux modifiés permettant d'augmenter un titre viral et diverses méthodes permettant d'augmenter de tels titres ainsi que des vecteurs d'expression capables d'améliorer de tels titres. L'invention concerne également des séquences isolantes fonctionnelles dérivées de l'isolant de la chromatine CHS4. L'invention concerne également des méthodes pour la correction génétique de maladies ou l'atténuation de symptômes associés, comme la drépanocytose ou la β-thalassémie.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)