Universitè Laval

Other

Cellectis

Jacques P Tremblay

Pierre Chapdelaine

Joël Rousseau

Zoe Coulombe

Victor-Alain Parent

Alain Garnier

Sébastien Goudenege

Nicolas B Huot

Carl Lebel

Frederic Cedrone

Basma Fatouma Benabdallah

Zoé Coulombe

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.

CRISPR-based methods and products for increasing frataxin levels and uses thereof

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.

Reduction of amyloid beta peptide production via modification of the APP gene using the CRISPR/Cas system

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.

Methods and products for increasing frataxin levels and uses thereof

A lighting structure for mounting a strip of LED's comprising a base member () having a channel () formed therein for receiving the strip () of LED's, a first member () of an attachment system extending along a portion of the base member (), and a cover () having a second portion () of an attachment system, said second portion () of said attachment system mating with the first portion () of the attachment system to retain the cover () and base member () together and at least partially overlapping the channel to permit retention of the strip of LED's therein.

Lighting structure

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.

Culture medium for myoblasts, precursors thereof and derivatives thereof

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.

Myogenic differentiation of stem cells and uses thereof

The present invention relates to a method to treat a genetic disease in an individual caused by at least one frame shift or at least one non sense mutation in the human dystrophin gene comprising at least the step of bringing into contact at least one meganuclease enzyme, which recognizes and cuts a target site in the human dystrophin gene, with the genome of said individual under conditions wherein said at least one meganuclease recognizes and cleaves its target site in the human dystrophin gene. Said method applies also to a set of meganuclease enzymes, which each recognizes and cuts a different target site. The present invention also relates to a kit comprising, at least one meganuclease enzyme as defined above and medicament comprising said meganuclease.

Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations

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.

Treatment of Duchenne muscular dystrophy with myoblasts expressing dystrophin and treated to block myostatin signaling

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.

[object Object]

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.

Treatment of hereditary diseases with gentamicin

A method of pretreating healthy donor's myoblast cultures with growth or trophic factors like basic fibroblast growth factor (bFGF) on transplantation to subjects suffering of recessive myopathy like muscular dystrophy is disclosed and claimed. Recipient muscles show a higher percentage of functional cells, demonstrated by the higher incidence of dystrophin-positive fibers, and does not require previous preconditioning of recipient muscles by irradiation or toxin administration. Donor mouse myoblasts expressing the reporter gene .beta.- galactosidase were grown with 100 ng/ml bFGF during the last two days before injecting them in the left tibialis anterior (TA) muscles of recipient MHC-compatible mdx mice, an experimental animal model of muscular dystrophy. Myoblasts from the same primary cultures were also grown without bFGF and injected in the right TA muscles as control. The recipient mice were immunosuppressed with FK 506. Twenty-eight days after myoblast transplantation, the percentage of .beta.- galactosidase-positive fibers was significantly higher (more than a 4 fold increase) following culture with bFGF than without bFGF. Almost all .beta.-galactosidase-positive-fibers were also dystrophin positive.

Growing community of inventors

CA

Jacques P Tremblay