Ucl Business Plc

Deepak Raj

Amit Nathwani

There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional α-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.

Fabry disease gene therapy

There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional &#x3b1;-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.

There is described a new gene therapy approach for treating Wilson's disease in which a nucleic acid molecule is used which comprises a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO: 1. Also described are vectors comprising the nucleotide sequence and methods and uses thereof.

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London, United Kingdom

Deepak Raj