| GENETHON Patent applications |
| Patent application number | Title | Published |
|---|
| 20120077860 | Adeno-Associated Viral Vector for Exon Skipping in a Gene Encoding a Dispensable Domain Protein - The invention concerns an adeno-associated viral vector comprising:
| 03-29-2012 |
| 20100266551 | ADENO-ASSOCIATED VIRAL VECTORS FOR THE EXPRESSION OF DYSFERLIN - The present invention relates to a composition comprising: a first adeno-associated viral (AAV) vector comprising: i) a 5′ITR (Inverted Terminal Repeat) sequence of AAV; ii) a portion of gene placed under the control of a promoter; iii) a sequence comprising a splice donor site; iv) a 3′ITR sequence of AAV; and/or a second adeno-associated viral (AAV) vector comprising; v) a 5′ITR (Inverted Terminal Repeat) sequence of AAV; vi) a sequence comprising a splice acceptor site; vii) a portion of gene; viii) a 3′ITR sequence of AAV. The combination of the portions of gene carried by the first and second AAV vectors comprises an open reading frame which encodes a functional dysferlin. In addition, the combination of the sequence comprising the splice donor site and the sequence comprising the splice acceptor site contains all the elements necessary for the splicing, advantageously derived from a natural intron of the dysferlin gene. | 10-21-2010 |
| 20100010036 | DRUGS FOR THE TREATMENT OF SARCOGLYCANOPATHIES - Inhibitors of the endoplasmic reticulum associated degradation (ERAD) pathway, particularly inhibitors of mannosidase I, are used for the preparation of a medicinal product intended to treat sarcoglycanopathies. | 01-14-2010 |
| 20090220468 | Specific CD4+CD25+ Regulatory T Cells for Haematopoietic Cell Transplantation and Immune Tolerance - A pharmaceutical composition comprising CD4+ CD25+ regulatory T cells specific for at least one minor histocompatibility antigen, and stem cells, advantageously haematopoietic, carrying at least the antigen can be used as a medicament for increasing the immune tolerance of a histocompatible host. | 09-03-2009 |
