The present invention provides novel dystrophin mini/micro-genes that retain the essential biological functions of a full-length dystrophin gene.
The present invention also provides compositions comprising mini-dystrophin peptides, and methods for expressing mini-dystrophin peptides in target cells.
The present invention also provides methods of treating disease by co-administration of nucleic cid sequences encoding Igf-1 and dystrophin or dystrophin-like proteins.
Dystrophin is encoded by a gene containing 79 exons responsible for producing a protein, called dystrophin.
A mutation in the dystrophin gene which codes for the protein called dystrophin causes Duchenne.
The present invention relates to compositions and methods for expressing mini-dystrophin peptides.
Expression of the integrin αBX2 polypeptide in muscle cells results in better physical condition in a patient or an animal lacking normal levels of dystrophin or dystrophin and utrophin.
Immunohistochemical processing of tissue which allows
In the absence of dystrophin, muscle cells deteriorate.
SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin transgene, called microdystrophin, to the body.
Utrophin is functionally and structurally similar to dystrophin.
In the absence of dystrophin, muscle cells deteriorate.
SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body.
Without dystrophin, muscles are delicate and are easily impaired.
Because dystrophin is missing, the muscle cells are damaged.
Without dystrophin, muscle cells become fragile and are easily damaged.
Normally, the dystrophin protein helps strengthen muscle fibres.
In Duchenne muscular dystrophy, dystrophin is almost totally absent; the less dystrophin that is produced, the worse the symptoms and etiology of the disease.
SGT-001 is systemically administered to deliver a synthetic dystrophin transgene, called microdystrophin, to the body.
a way to measure the amount of dystrophin in the muscle
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