Abstract: Methods and products (e.g., guide RNAs or pegRNA and recombinant proteins) are described for decreasing amyloidogenic A? peptide levels produced by a cell, or the aggregation of the A? peptide, as well as uses of such methods and products, for example for the treatment of Alzheimer's disease and/or age-related cognitive decline in a subject in need thereof.

Abstract: Methods of modifying a frataxin gene are disclosed, comprising removing some or all of endogenous GAA trinucleotide repeats within the frataxin gene, e.g., within an intron (e.g., intron 1) of the frataxin gene. The removal may be effected using a CRISPR/CAS nuclease system. Such modification may be used to increase frataxin expression in the cell, and also to treat a subject suffering from Friedreich ataxia. Reagents, kits and uses of the method are also disclosed, for example to modify a frataxin gene and to treat a subject suffering from Friedreich ataxia.

Abstract: Methods for modifying a dystrophin gene are disclosed, for restoring dystrophin expression within a cell having an endogenous frameshift or nonsense mutation within the dystrophin gene. The methods comprise introducing a first cut within an exon en or intron of the dystrophin gene creating a first exon end or intron end, wherein said first cut is located upstream of the endogenous frameshift or nonsense mutation; and introducing a second cut within an exon or intron of the dystrophin gene creating a second exon end or intron end, wherein said second cut is located downstream of the frameshift or nonsense mutation. Upon joining/ligation of said first and second exon ends or intron ends a hybrid exon or intron junction is created and dystrophin expression is restored, as the correct reading frame is restored. Reagents and uses of the method are also disclosed, for example to treat a subject suffering from muscular dystrophy.

Abstract: Methods and products (e.g., gRNAs, recombinant fusion proteins, frataxin targeting systems, compositions and kits) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom.

Abstract: Methods and products are described related to use of the CRISPR/Cas9 system to introduce a modification into an APP gene, such as guide RNAs and recombinant proteins, for decreasing amyloid beta peptide produced by a cell. Also described are uses of such methods and products for the treatment of Alzheimer's disease and/or age-related cognitive decline in a cell from a subject in need thereof.

Abstract: Methods of modifying a dystrophin gene are disclosed, for restoring dystrophin expression within a cell having an endogenous frameshift mutation within the dystrophin gene. The methods comprising introducing a first cut within an exon of the dystrophin gene creating a first exon end, wherein said first cut is located upstream of the endogenous frameshift mutation; and introducing a second cut within an exon of the dystrophin gene creating a second exon end, wherein said second cut is located downstream of the frameshift mutation. Upon joining/ligation of said first and second exon ends dystrophin expression is restored, as the correct reading frame is restored. Reagents and uses of the method are also disclosed, for example to treat a subject suffering from muscular dystrophy.

Abstract: Methods and products (e.g., gRNAs, recombinant fusion proteins, frataxin targeting systems, compositions and kits) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom.

Abstract: Methods and products (e.g., recombinant proteins) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom.

Abstract: Methods and products related to genome editing using the CRISPR/Cas9 system to introduce an A673T substitution into an APP gene, such as guide RNAs and recombinant proteins, are described for decreasing APP levels produced by a cell, and use for the treatment of Alzheimer's disease and/or age-related cognitive decline in a cell from a subject in need thereof.

Abstract: Methods and products (e.g., recombinant proteins) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom.

Abstract: This application relates to a serum-free culture medium that enables the proliferation of cell of the myogenic lineage while maintaining their ability to differentiate into functional muscle cells. Also contemplated herewith are method of culturing cells of the myogenic lineages and uses of the cultured cells for the treatment or the alleviation of symptoms of a muscular deficiencies.

Abstract: Disclosed are methods of differentiating stem cells into muscle cells by growing the cells in a myogenic culture medium. The differentiated cells can be used as a source of cells for transplantation in a patient in need thereof.

Abstract: Methods and products (e.g., recombinant proteins) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom.

Abstract: Disclosed are methods of differentiating stem cells into muscle cells by growing the cells in a myogenic culture medium. The differentiated cells can be used as a source of cells for transplantation in a patient in need thereof.

Abstract: This application relates to a serum-free culture medium that enables the proliferation of cell of the myogenic lineage while maintaining their ability to differentiate into functional muscle cells. Also contemplated herewith are method of culturing cells of the myogenic lineages and uses of the cultured cells for the treatment or the alleviation of symptoms of a muscular deficiencies.

Abstract: The invention relates to methods and materials for enhancing muscle mass or for the treatment of muscle disease in a subject, comprising introducing a cell which has a lower than normal level of myostatin signalling, into the subject.

Abstract: Methods of pre-treating healthy donor's myoblast cultures with growth or trophic factors on transplantation into subjects suffering from myopathic conditions such as muscular dystrophy. Compositions comprising myoblasts and fusion-promoting metalloproteases can be transplanted. Alternatively, myoblasts can be transplanted along with an agent inducing the expression of a fusion-promoting metalloprotease, or a composition comprising genetically-modified myoblasts capable of expressing a fusion-promoting metalloprotease can be transplanted.

Abstract: A method of pretreating healthy donor's myoblast cultures with growth or trophic factors like basic fibroblast growth factor (bFGF) and with concanavalin A on transplantation to subjects suffering of myopathy like muscular dystrophy is disclosed and claimed. Recipient muscles show a higher percentage of functional cells, a four-fold increase, demonstrated by the higher incidence of dystrophin-positive fibers, and does not require previous preconditioning of recipient muscles by irradiation or toxin administration. The recipient subjects were immunosuppressed with FK 506. When growing myoblasts with 20 &mgr;g/ml concanavalin A or 100 ng/ml TPA for two to four days, migration of donor cells in recipient tissue was increased by 3-4 fold.

Abstract: The present invention relates to a method for preconditioning healthy donor's myoblasts in vitro before transplantation thereof in compatible patients suffering of recessive myopathies, particularly of muscular dystrophy. This in vitro preconditioning improves the success of the transplantation while not requiring an in vivo preconditioning of the patient's muscle by irradiation or by administering muscular toxin. The invention further relates to compositions comprising such preconditioned myoblasts. The preconditioning comprises pre-treating the transplanted myoblasts with human fibroblast growth factor (bFGF). The transplantation is made with the whole culture along with bFGF. A concentration of 100 ng/ml bFGF improved the myoblasts fusion by a four fold average.

Abstract: This invention relates to a method of treating an inherited disease due to a point mutation producing a stop codon by administering an effective dose of an aminoglycoside antibiotic or a derivative thereof. Mdx mouse, which is an animal model for Duchenne muscular dystrophy, has been successfully treated with intramuscularly administered 1 and 5 mg gentamicin, which had for effect to suppress the premature stop mutation by inserting an amino acid at the stop codon. Dystrophin positive muscle fibers not different in number from those of normal mouse were detected at the dose of 5 mg gentamicin.