Abstract: The present disclosure is in the field of gene therapy, in particular for the treatment of cholestatic disease. More specifically, the present invention relates to a minimal bile acid inducible promoter and its use for gene therapy in cholestatic disease.

Abstract: AG5 andrographolide derivative for use in the treatment of the inflammatory reaction caused by a cytokine storm, particularly produced by CoViD-19, bacteria with superantigens or by CAR-T, TIL or BiTE cell therapies.

Abstract: The present disclosure relates to viral particles for use in treating tauopathies, particularly Alzheimer's disease, by gene therapy. More specifically, the present invention relates to a viral particle for use in treating tauopathies by gene therapy in a subject in need thereof, said viral particle comprising a nucleic acid construct including a transgene encoding a glucocerebro sidase.

Abstract: The present disclosure relates to gene therapy vector for use in the treatment of cerebro tendinous xanthomatosis. More specifically, the present invention relates to a nucleic acid construct comprising liver specific promoter operably linked to a nucleic acid sequence encoding for the sterol 27-hydroxylase for the treatment of CTX.

Abstract: The present disclosure relates to viral particles for use in treating synucleinopathies, particularly sporadic Parkinson Diseases by gene therapy. More specifically, the present invention relates to a viral particle for use in treating synucleinopathy by gene therapy in a subject in need thereof, said viral particle comprising a nucleic acid construct including a transgene encoding a glucocerebrosidase.

Abstract: The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.

Abstract: A gene therapy vector is used in the treatment of progressive familial intrahepatic cholestasis type 2. More specifically, an adeno-associated virus vector includes codon-optimized sequence encoding for the BSEP for the treatment of PFIC2.

Abstract: The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.

Abstract: Gene therapy vector is for use in the treatment of progressive familiar intrahepatic cholestasis type 3. More specifically, an adeno-associated virus vector includes codon-optimized sequence encoding for the MDR3 isoform A for the treatment of PFIC3.

Abstract: The invention relates to a nucleic acid construct carrying ATP7B protein, an expression vector and a viral particle comprising the nucleic acid construct, and their use for treatment of Wilson's disease and other conditions caused by a deficiency or dysfunction of ATP7B protein. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.

Abstract: A field of gene therapy and engineering of viral vectors for use in gene therapy. More specifically, it is disclosed herein adeno-associated virus vectors and expression cassettes comprising S/MAR sequences of c-Myc or IFN-? for the treatment of liver diseases, notably in neonates.

Abstract: The invention relates to a nucleic acid construct carrying ATP7B protein, an expression vector and a viral particle comprising the nucleic acid construct, and their use for treatment of Wilson's disease and other conditions caused by a deficiency or dysfunction of ATP7B protein. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.

Abstract: The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.

Abstract: The present invention is generally related to the prevention of leishmaniasis in animals, particularly infection caused by Leishmania sp., based on the use of Leishmania infantum P36 protein or an immunogenic fragment of the latter, or involving an expression system for the mentioned protein or fragment—optionally in combination with a compound stimulating the production of a Th1-type cellular immune response—and comprising various vaccination protocols in application to Leishmania sp. based on the mentioned vaccine.

Abstract: The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.

Abstract: The application relates to gene constructs for inducible hepato-specific expression of polynucleotides of interest in response to an inducer agent, said constructs comprising (i) an inducible bi-directional operator-promoter with at least one responsive element to said inducer agent flanked by two hepato-specific promoters acting in divergent manner, (ii) a first nucleotide sequence encoding a transactivator which may be activated by said inducer agent operatively coupled to the first hepato-specific promoter and (iii) a second nucleotide sequence operatively coupled to the second hepato-specific promoter, wherein the promoters are induced as a consequence of the binding of the transactivator to the operator region of the operator-promoter in the presence of the inducer agent.

Abstract: The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.

Abstract: The present invention relates to methods and compositions for augmenting an immunogenicity of an antigen in a mammal, comprising administering said antigen together with an adjuvant composition that includes glycosylceramide, preferably ?-galactosylceramide (?-GalCer). According to the present invention, the use of glycosylceramide as an adjuvant is attributed at least in part to the enhancement and/or extension of antigen-specific Th1-type responses, in particular, CD8+ T cell responses. The methods and compositions of the present invention can be useful for prophylaxis and treatment of various infectious and neoplastic diseases.

Abstract: The present invention is generally related to the prevention of leishmaniasis in animals, particularly infection caused by Leishmania sp., based on the use of Leishmania infantum P36 protein or an immunogenic fragment of the latter, or involving an expression system for the mentioned protein or fragment—optionally in combination with a compound stimulating the production of a Th1-type cellular immune response—and comprising various vaccination protocols in application to Leishmania sp. based on the mentioned vaccine.

Abstract: The present invention relates to methods and compositions for augmenting an immunogenicity of an antigen in a mammal, comprising administering said antigen together with an adjuvant composition that includes glycosylceramide, preferably &agr;-galactosylceramide (&agr;-GalCer). According to the present invention, the use of glycosylceramide as an adjuvant is attributed at least in part to the enhancement and/or extension of antigen-specific Th1-type responses, in particular, CD8+ T cell responses. The methods and compositions of the present invention can be useful for prophylaxis and treatment of various infectious and neoplastic diseases.