Ucl Business Plc

St. Jude Children's Research Hospital

Thrombosis Research Institute

University of California

Adrian Thrasher

Amit Nathwani

Edward Tuddenham

John T Gray

Andrew Davidoff

Natalie Ward

John McVey

John Dixon Gray

Bobby Gaspar

John Mcvey

Donald B Kohn

Michael Antoniou

Claudia Montiel-Equihua

An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocyte biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.

Optimised coding sequence and promoter

An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes arc alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.

The present invention relates to the prevention and/or treatment of ADA-SCID, in a patient.

Method for treating adenosine deaminase severe combined imunodeficiency

The present invention relates to the prevention and/or treatment of lysosomal storage diseases in a patient.

Treatment of lysosomal storage diseases

An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.

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London, United Kingdom

Adrian Thrasher