摘要:
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
摘要:
A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
摘要:
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
摘要:
The present invention concerns a method for the identification of new disease biomarkers, e.g. from cancer or diabetes mellitus patients, in undepleted serum by a novel multi-denaturation method and use of said cancer biomarkers for the diagnosis and treatment of cancer.
摘要:
The present invention relates to modified nucleic acid sequences coding for coagulation Factors, in particular human FV and human Factor VIII and their derivatives with improved stability, recombinant expression vectors containing such nucleic acid sequences, host cells transformed with such recombinant expression vectors, recombinant polypeptides and derivatives which do have biological activities of the unmodified wild type protein but having improved stability and processes for the manufacture of such recombinant proteins and their derivatives. The invention also covers a transfer vector for use in human gene therapy, which comprises modified DNA sequences.
摘要:
Dithiocarbamate, particularly tetraethylthiuram disulfide strongly inhibits the growth of cancer cells of a variety of cell types. Such inhibitory effect is enhanced by heavy metal ions such as copper ions, cytokines and ceruloplasmin. A method is presented for using tetraethylthiuram disulfide to reduce tumor growth, and to potentiate the effect of other anticancer agents.
摘要:
A neuroprotective composition for protecting neuronal cells against oxidative stress and methods for using and preparing the same. More particularly, the neuroprotective composition of the invention comprises a therapeutically effective amount of ceruloplasmin or a functional derivative thereof. The neuroprotective composition is characterized in that it protects neuronal cells from reactive oxygen species such as •O2- and •OH. In a preferred embodiment, the neuroprotective composition further comprises an antioxidant consisting of catalase or of an amphiphilic physiological antioxidative solution comprising a mixture of pyruvate, antioxidant, and lipid(s) such as fatty acids. The neuroprotective composition could be used for the treatment of brain trauma, brain or cerebrovascular ischemia, neurodegenerative diseases, poisoning of neuronal cells, the diminution of drugs side effects and for preservation of neuronal grafts.
摘要:
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
摘要:
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
摘要:
Described herein are compositions and methods for treating Friedreich's Ataxia (FRDA). In some aspects, mutant forms of frataxin which are resistant to ubiquitination are provided. In some aspects, pharmaceutical compositions comprising mutant frataxin are provided. In further aspects, methods of using mutant frataxin are provided.