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 US17/596,476 priority patent/US20220175966A1/en
Priority to CN202080042904.4A priority patent/CN113950533A/zh
Priority to EP20730331.4A priority patent/EP3983550A1/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
US17/596,476 US20220175966A1 (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
EP20730331.4A EP3983550A1 (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
CA3156506A1 (en) * 2019-10-30 2021-05-06 Amicus Therapeutics, Inc. Recombinant cdkl5 proteins, gene therapy and production methods
EP4117735A4 (en) * 2020-03-09 2024-04-10 University of Massachusetts Gene replacement therapy for foxg1 syndrome
JP2023539574A (ja) * 2020-08-17 2023-09-15 マサチューセッツ インスティテュート オブ テクノロジー Shank3遺伝子治療アプローチ
MX2023006445A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
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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WO2002097056A2 (en) * 2001-05-31 2002-12-05 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
US20060239966A1 (en) * 2003-10-20 2006-10-26 Tornoee Jens In vivo gene therapy of parkinson's disease
CA3205555A1 (en) * 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
EP4154914B1 (en) * 2015-05-07 2024-08-28 University of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
BR112018077225A2 (pt) * 2016-06-28 2019-04-09 Alma Mater Studiorum - Università di Bologna proteínas de fusão tatk-cdkl5, composições, formulações e uso das mesmas
KR20200090889A (ko) * 2017-11-30 2020-07-29 아미쿠스 세라퓨틱스, 인코포레이티드 Cdkl5 발현 변이체 및 cdkl5 융합 단백질

Non-Patent Citations (27)

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Title
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J. M. POWERSX. L. CHANGZ. SONGH. NAKAI: "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
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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
EP3983550A1 (en) 2022-04-20
US20220175966A1 (en) 2022-06-09
CN113950533A (zh) 2022-01-18
WO2020250081A1 (en) 2020-12-17

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