摘要:
There are provided compositions and methods for modulating stem cell division, in particular, division symmetry. It has been demonstrated that Wnt7a polypeptide fragments promoting symmetrical expansion of stem cells. The compositions and methods of the invention are useful, for example, in modulating stem cell division symmetry in vitro, ex vivo, and in vivo, in replenishing and expanding the stem cell pool, and in promoting the formation, maintenance, repair and regeneration of tissue.
摘要:
In certain aspects, the present invention provides compositions and methods for inducing utrophin expression in muscle with an ActRIIB protein as therapy for muscular dystrophy. The present invention also provides methods of screening compounds that modulate activity of an ActRIIB protein and/or an ActRIIB ligand.
摘要:
The invention relates to synthetic polynucleotides encoding fukutin related protein (FKRP). The invention further relates to nucleic acid constructs comprising the synthetic polynucleotides and methods of using these synthetic polynucleotides to treat dystroglycanopathy disorders.
摘要:
In certain aspects, the present invention provides compositions and methods for inducing utrophin expression in muscle with an ActRIIB protein as therapy for muscular dystrophy. The present invention also provides methods of screening compounds that modulate activity of an ActRIIB protein and/or an ActRIIB ligand.
摘要:
The present invention provides compositions and methods for using correctional RNA, with a proteinaceous carrier, to stimulate respiration of the cells, tissues, organs or the whole organism of normal or diseased subjects. In one embodiment of the invention, the signal-tagged correctional RNA is one or more protein-coding RNAs (pcRNA) that encode one or more of human mitochondrial proteins. The invention specifically provides a broad-spectrum formulation applicable to a wide variety of disorders that are associated with mitochondrial mutations.
摘要:
The present invention relates to a nucleic acid molecule comprising a nucleotide sequence codifying for a protein characterized in having at least four zinc finger domains and in modulating the transcription of the utrophin gene by binding to a nucleotidic sequence comprised in a utrophin gene untranscribed regulatory sequence. Protein codified thereby and uses thereof.
摘要:
The invention relates to a gene-therapeutic nucleic acid construct containing a regulatory nucleic acid sequence of the 5´-end of the myosin light chain 2 (MLC-2) heart gene. The regulatory nucleic acid sequence in question is functionally connected to a nucleic acid which codes for a therapeutically active gene product, antisense nucleic acid or ribozyme. Also disclosed is a process for producing the construct and its use in gene therapy for treating heart disorders.