Patents Assigned to ASSOCIATION INSTITUT DE MYOLOGIE
  • Modified U7 snRNAs for treatment of neuromuscular diseases
    Patent number: 9080170
    Abstract: The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified U7 snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.
    Type: Grant
    Filed: March 17, 2011
    Date of Patent: July 14, 2015
    Assignee: ASSOCIATION INSTITUT DE MYOLOGIE
    Inventors: Luis Garcia, Denis Furling, Cyriaque Beley, Thomas Voit
  • CAPSID-FREE AAV VECTORS, COMPOSITIONS, AND METHODS FOR VECTOR PRODUCTION AND GENE DELIVERY
    Publication number: 20140107186
    Abstract: An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.
    Type: Application
    Filed: March 12, 2012
    Publication date: April 17, 2014
    Applicants: ASSOCIATION INSTITUT DE MYOLOGIE, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
    Inventors: Luis Garcia, Cyriaque Beley, Thomas Voit, Robert Michael Kotin
  • METHODS OF INCREASING EFFICIENCY OF VECTOR PENETRATION OF TARGET TISSUE
    Publication number: 20130189225
    Abstract: Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP.
    Type: Application
    Filed: March 22, 2011
    Publication date: July 25, 2013
    Applicants: ASSOCIATION INSTITUT DE MYOLOGIE, UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6), CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, GENETHON
    Inventors: Thomas Voit, Luis Garcia, Jérôme Denard, Fedor Svinartchouk
  • NUCLEIC ACID MOLECULES AND METHODS FOR EXCHANGING EXON(S) BY TRANSSPLICING
    Publication number: 20130065948
    Abstract: The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated simple or double trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and to mediate a simple or double trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA).
    Type: Application
    Filed: October 8, 2010
    Publication date: March 14, 2013
    Applicants: ASSOCIATION INSTITUT DE MYOLOGIE, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), INTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM), UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
    Inventors: Luis GARCIA, Stephanie LORAIN
  • MODIFIED U7 SNRNAS FOR TREATMENT OF NEUROMUSCULAR DISEASES
    Publication number: 20130045538
    Abstract: The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.
    Type: Application
    Filed: March 17, 2011
    Publication date: February 21, 2013
    Applicant: ASSOCIATION INSTITUT DE MYOLOGIE
    Inventors: Luis Garcia, Denis Furling, Cyriaque Beley, Thomas Voit
  • NUCLEIC ACID MOLECULES AND METHODS FOR EXCHANGING EXON(S) BY TRANSSPLICING
    Publication number: 20120245220
    Abstract: The present invention provided methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated simple or double trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and to mediate a simple or double trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (Chimeric RNA).
    Type: Application
    Filed: October 8, 2010
    Publication date: September 27, 2012
    Applicants: UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6), ASSOCIATION INSTITUT DE MYOLOGIE
    Inventors: Luis Garcia, Stéphanie Lorain