摘要:
The invention relates to a composition including (1) an anionic macromolecule except nucleic acids, (2) a cationic lip, and (3) a nucleic acid having a size lower than or equal to 200 nucleotides, such as an interference RNA, and to the use thereof in gene therapy.
摘要:
The present invention relates, generally, to improved methods of delivering a biologically active agent, in particular a therapeutic or prophylactic nucleic acid, to the ocular sphere of a subject comprising administering said agent to the ciliary body tissue(s) or cells and/or to the extra-ocular muscle tissue or cells. More particularly, the invention relates to devices, their uses, notably in gene therapy, and to methods for treating pathologies of the ocular sphere by specific ciliary body tissue(s) or cells and/or extra-ocular muscle or cells administration of a therapeutic product and transfer thereof into the ocular tissue to be treated. This invention also relates to pharmaceutical compositions comprising the product in a form suitable for ciliary body tissue(s) or cells and/or extra-ocular muscle or cells administration, their preparation and uses.
摘要:
The present invention relates, generally, to improved methods of delivering a biologically active agent, in particular a therapeutic or prophylactic nucleic acid, to the ocular sphere of a subject comprising administering said agent to the ciliary body tissue(s) or cells and/or to the extra-ocular muscle tissue or cells. More particularly, the invention relates to devices, their uses, notably in gene therapy, and to methods for treating pathologies of the ocular sphere by specific ciliary body tissue(s) or cells and/or extra-ocular muscle or cells administration of a therapeutic product and transfer thereof into the ocular tissue to be treated. This invention also relates to pharmaceutical compositions comprising the product in a form suitable for ciliary body tissue(s) or cells and/or extra-ocular muscle or cells administration, their preparation and uses.
摘要:
The present invention relates to gelling compositions, which changes from liquid state to gel state in function of temperature comprising: at least a poloxamer or mixture of poloxamers; at least a gelling agent; and at least an anticancer agent. Said compositions are advantageously used for local administration of an anticancer agent. Said compositions are useful for size-reduction of a tumour before surgical removal of said tumour, for preventing tumour recurrence after surgical removal of a tumour, and/or treating small tumours. They are therefore useful for the treatment of cancer, preferably a cancer of a wall of the digestive system or a gynaecologic cancer. The present invention also relates to a method for preparing said gelling compositions.
摘要:
The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.
摘要翻译:本发明涉及一种用于治疗患有CEP290基因突变c.2991 + 1655A> G的患者的Leber先天性黑素瘤的方法,包括向所述患者施用至少一种与核酸序列互补的反义寡核苷酸的步骤 这是防止插入到突变体c.2291 + 1655A> G CEP290 mRNA中的神经外显子的剪接所必需的。
摘要:
Multimodal optical and magnetic resonance imaging methods based on the use of persistent luminescence nanoparticles. Use of mesoporous persistent luminescence "core-shell" complexes for theranostic applications.
摘要:
The invention concerns an improved method for transferring in vivo into the cells of striped muscles nucleic acids or nucleic acids combined with products for enhancing the efficacy of such transfers. The invention also concerns the combination of a nucleic acid and the transfer method for use in gene therapy.
摘要:
The invention relates to a mutated cell such as a bacteria or yeast in which the thyA gene coding thymidylate synthase includes a nonsense codon, preferably the amber codon, said nonsense codon replacing a codon coding an amino acid and inducing the interruption of thyA gene translation and the auxotropy of the cell for the thymidine. Advantageously, the endA gene coding the endonuclease 1 and/or the recA gene coding the recombinase is inactivated in said mutated cell. The invention also relates to an expression plasmid including a transgene and a sequence of a suppressing ARM structural gene containing an anticodon that can be paired with the nonsense codon of the thyA gene and is specific of an amino acid capable of restoring the translation of the mutated thyA gene and thereby obtaining a protein of the wild or mutated type having a thymidylate synthase activity. The invention also relates to a method for the multiplication of the expression plasmid.