IT201900008877A1 - Nuovi costrutti per terapia genica - Google Patents

Nuovi costrutti per terapia genica

Info

Publication number
IT201900008877A1
IT201900008877A1 IT102019000008877A IT201900008877A IT201900008877A1 IT 201900008877 A1 IT201900008877 A1 IT 201900008877A1 IT 102019000008877 A IT102019000008877 A IT 102019000008877A IT 201900008877 A IT201900008877 A IT 201900008877A IT 201900008877 A1 IT201900008877 A1 IT 201900008877A1
Authority
IT
Italy
Prior art keywords
gene therapy
new buildings
buildings
new
therapy
Prior art date
Application number
IT102019000008877A
Other languages
English (en)
Inventor
Elisabetta Ciani
Hiroyuki Nakai
Stefania Trazzi
Claudia Fuchs
Original Assignee
Univ Bologna Alma Mater Studiorum
Univ Oregon Health & Science
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Bologna Alma Mater Studiorum, Univ Oregon Health & Science filed Critical Univ Bologna Alma Mater Studiorum
Priority to IT102019000008877A priority Critical patent/IT201900008877A1/it
Priority to PCT/IB2020/055165 priority patent/WO2020250081A1/en
Priority to EP20730331.4A priority patent/EP3983550A1/en
Priority to CN202080042904.4A priority patent/CN113950533A/zh
Priority to US17/596,476 priority patent/US20220175966A1/en
Publication of IT201900008877A1 publication Critical patent/IT201900008877A1/it

Links

IT102019000008877A 2019-06-13 2019-06-13 Nuovi costrutti per terapia genica IT201900008877A1 (it)

Priority Applications (5)

Application Number Priority Date Filing Date Title
IT102019000008877A IT201900008877A1 (it) 2019-06-13 2019-06-13 Nuovi costrutti per terapia genica
PCT/IB2020/055165 WO2020250081A1 (en) 2019-06-13 2020-06-01 New gene therapy constructs
EP20730331.4A EP3983550A1 (en) 2019-06-13 2020-06-01 New gene therapy constructs
CN202080042904.4A CN113950533A (zh) 2019-06-13 2020-06-01 新基因疗法构建体
US17/596,476 US20220175966A1 (en) 2019-06-13 2020-06-01 New gene therapy constructs

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
IT102019000008877A IT201900008877A1 (it) 2019-06-13 2019-06-13 Nuovi costrutti per terapia genica

Publications (1)

Publication Number Publication Date
IT201900008877A1 true IT201900008877A1 (it) 2020-12-13

Family

ID=68426584

Family Applications (1)

Application Number Title Priority Date Filing Date
IT102019000008877A IT201900008877A1 (it) 2019-06-13 2019-06-13 Nuovi costrutti per terapia genica

Country Status (5)

Country Link
US (1) US20220175966A1 (it)
EP (1) EP3983550A1 (it)
CN (1) CN113950533A (it)
IT (1) IT201900008877A1 (it)
WO (1) WO2020250081A1 (it)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230043046A1 (en) * 2019-10-30 2023-02-09 Amicus Therapeutics, Inc. Recombinant CDKL5 Proteins, Gene Therapy and Production Methods
US12497612B2 (en) 2020-03-09 2025-12-16 University Of Massachusetts Gene replacement therapy for FOXG1 syndrome
CN116406305A (zh) * 2020-08-17 2023-07-07 麻省理工学院 Shank3基因治疗方法
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
WO2024178401A1 (en) * 2023-02-24 2024-08-29 Massachusetts Institute Of Technology Shank3 gene therapy approaches
CN120648654A (zh) * 2024-03-13 2025-09-16 深圳太力生物技术有限责任公司 重组细胞的制备方法、重组细胞及其应用
EP4647439A1 (en) * 2024-05-09 2025-11-12 Alma Mater Studiorum - Università di Bologna Non-pathogenic bacteria for delivery of ptd-fusion proteins

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US8067156B2 (en) * 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
CN1897977A (zh) * 2003-10-20 2007-01-17 Ns基因公司 帕金森氏病的体内基因治疗
CN114395559A (zh) * 2014-04-25 2022-04-26 吉尼松公司 高胆红素血症的治疗
EP3291843B1 (en) * 2015-05-07 2023-03-22 University of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
EP3475418A4 (en) * 2016-06-28 2020-03-04 Alma Mater Studiorum -Universita' di Bologna TATK-CDKL5 FUSION PROTEINS, COMPOSITIONS, FORMULATIONS AND THEIR USE
JP2021505135A (ja) * 2017-11-30 2021-02-18 アミカス セラピューティックス インコーポレイテッド Cdkl5発現変異体及びcdkl5融合タンパク質

Non-Patent Citations (27)

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Title
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N. BAHI-BUISSONT. BIENVENU: "CDKL5-Related Disorders: From Clinical Description to Molecular Genetics", MOLECULAR SYNDROMOLOGY, vol. 2, 2012, pages 137 - 152
O. H. LOWRYN. J. ROSEBROUGHA. L. FARRR. J. RANDALL: "Protein measurement with the Folin phenol reagent", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 193, 1951, pages 265 - 275, XP000196391
POWERS JMCHANG XLSONG ZNAKAI H: "A Quantitative Dot Blot Assay for AAV Titration and Its Use for Functional Assessment of the Adeno-associated Virus Assembly-activating Proteins", J VIS EXP, 2018, pages 12
S. BASTIANINI ET AL.: "Accurate discrimination of the wake-sleep states of mice using non-invasive whole-body plethysmography", SCIENTIFIC REPORTS, vol. 7, 2017, pages 41698
S. J. GRAY ET AL.: "Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors", HUM GENE THER, vol. 22, 2011, pages 1143 - 1153, XP055198141, DOI: 10.1089/hum.2010.245
STEFANIA TRAZZI ET AL: "CDKL5 protein substitution therapy rescues neurological phenotypes of a mouse model of CDKL5 disorder", HUMAN MOLECULAR GENETICS, vol. 27, no. 9, 20 February 2018 (2018-02-20), pages 1572 - 1592, XP055653212, ISSN: 0964-6906, DOI: 10.1093/hmg/ddy064 *
STEFANIA TRAZZI ET AL: "CDKL5 protein substitution therapy rescues neurological phenotypes of a mouse model of CDKL5 disorder.", HUMAN MOLECULAR GENETICS, 20 February 2018 (2018-02-20), pages 1 - 12, XP055653227, Retrieved from the Internet <URL:https://academic.oup.com/hmg/article/27/9/1572/4892297#supplementary-data> [retrieved on 20191216] *
STEVEN J. GRAY ET AL: "Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors", HUMAN GENE THERAPY, vol. 22, no. 9, 1 September 2011 (2011-09-01), pages 1143 - 1153, XP055198141, ISSN: 1043-0342, DOI: 10.1089/hum.2010.245 *
V. LO MARTIRE ET AL.: "CDKL5 deficiency entails sleep apnoea's in mice", JOURNAL OF SLEEP RESEARCH, vol. 26, 2017, pages 495 - 497
XIAN-CANG MA ET AL: "Intranasal Delivery of Recombinant AAV Containing BDNF Fused with HA2TAT: a Potential Promising Therapy Strategy for Major Depressive Disorder", SCIENTIFIC REPORTS, vol. 6, no. 1, 3 March 2016 (2016-03-03), XP055653418, DOI: 10.1038/srep22404 *

Also Published As

Publication number Publication date
WO2020250081A1 (en) 2020-12-17
CN113950533A (zh) 2022-01-18
US20220175966A1 (en) 2022-06-09
EP3983550A1 (en) 2022-04-20

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