AU2001261063A1 - Dna sequences encoding dystrophin minigenes and methods of use thereof - Google Patents

Dna sequences encoding dystrophin minigenes and methods of use thereof

Info

Publication number
AU2001261063A1
AU2001261063A1 AU2001261063A AU6106301A AU2001261063A1 AU 2001261063 A1 AU2001261063 A1 AU 2001261063A1 AU 2001261063 A AU2001261063 A AU 2001261063A AU 6106301 A AU6106301 A AU 6106301A AU 2001261063 A1 AU2001261063 A1 AU 2001261063A1
Authority
AU
Australia
Prior art keywords
dystrophin
present
minigenes
provides
dystrophin minigenes
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2001261063A
Inventor
Xiao Xiao
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of AU2001261063A1 publication Critical patent/AU2001261063A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Abstract

The present invention provides a series of novel dystrophin minigenes that retain the essential biological functions. The expression of the dystrophin minigenes may be controlled by a regulatory element along with a small polyadenylation signal. The entire gene expression cassettes may be readily packaged into a viral vector, preferably an AAV vector. The present invention further defines the minimal functional domains of dystrophin and provides ways to optimize and create new versions of dystrophin minigenes. Finally, the present invention provides a method of treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
AU2001261063A 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof Abandoned AU2001261063A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US20077700P 2000-04-28 2000-04-28
US60/200,777 2000-04-28
PCT/US2001/013677 WO2001083695A2 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Publications (1)

Publication Number Publication Date
AU2001261063A1 true AU2001261063A1 (en) 2001-11-12

Family

ID=22743139

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001261063A Abandoned AU2001261063A1 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Country Status (9)

Country Link
US (2) US7001761B2 (en)
EP (1) EP1287125B1 (en)
CN (1) CN100422320C (en)
AT (1) ATE437944T1 (en)
AU (1) AU2001261063A1 (en)
CA (1) CA2407309C (en)
DE (1) DE60139394D1 (en)
ES (1) ES2330615T3 (en)
WO (1) WO2001083695A2 (en)

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WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
EA035882B1 (en) 2013-03-14 2020-08-27 Сарепта Терапьютикс, Инк. Antisense oligonucleotides inducing exon skipping for treating muscular dystrophy
BR112015022998A2 (en) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc improved compositions for treating muscular dystrophy
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
MA45477A (en) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital ADENOASSOCIATED VIRUS VECTOR VECTOR MICROARN-29 AND MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
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CA3099958A1 (en) * 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
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Also Published As

Publication number Publication date
US20030171312A1 (en) 2003-09-11
CN100422320C (en) 2008-10-01
WO2001083695A2 (en) 2001-11-08
ES2330615T3 (en) 2009-12-14
CA2407309C (en) 2011-08-02
CN1439051A (en) 2003-08-27
DE60139394D1 (en) 2009-09-10
US7510867B2 (en) 2009-03-31
US20060073586A1 (en) 2006-04-06
EP1287125A4 (en) 2005-04-27
US7001761B2 (en) 2006-02-21
ATE437944T1 (en) 2009-08-15
WO2001083695A3 (en) 2002-03-07
EP1287125A2 (en) 2003-03-05
EP1287125B1 (en) 2009-07-29
CA2407309A1 (en) 2001-11-08
WO2001083695A9 (en) 2002-12-12

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