1996 – 2000

2000

Dr James Edmond Wraith £186,000: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
Dr Rob Wynn, Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England, Dr Bryan Winchester
Institute of Child Health, London, England
Year one of a three-year two-centre programme grant:

a) To establish the molecular basis of novel mutations found in patients with MPS I, Hurler disease, and MPS III, Sanfilippo disease
b) To develop gene therapy for MPS diseases using herpes virus vectors
c) To develop gene therapy using bone marrow cells and to investigate how the patient tolerates these new cells
d) To produce antibodies for use in gene therapy

The Mucopolysaccharidoses (MPS) result from genetic defects in enzymes involved in the turnover of large molecules call glycosaminoglycans (GAGs) which are important in maintaining the body’s skeleton and brain function. Research indicated that it might be possible to replace the defective enzyme in MPS by putting a good copy of the gene that is responsible for the synthesis of the defective enzyme into the patient’s cells – gene therapy. The major problems with gene therapy are rejection of the replacement protein by the immune system of the body, getting the enzyme into all tissues, particularly the brain, and the short life of the replacement gene or enzyme. These projects sought to solve these problems.

Dr James Edmond Wraith £31,000: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
To fund 50% of the final year of a three-year appointment for a second Consultant Paediatrician to specialise in the diagnosis and clinical management of MPS children and their families throughout the United Kingdom.

Dr Guy Besley £39,016: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
Final year of a three-year grant to extend the range of mutation analyses offered to MPS families by employing a dedicated member of staff to undertake these investigations.

1999

Dr James Edmond Wraith £28,000; Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
To fund 50% of year two of a three-year appointment for a second Consultant Paediatrician to specialise in the diagnosis and clinical management of MPS children and their families throughout the United Kingdom.

Dr Guy Besley £37,918: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
Year two of a three-year grant to extend the range of mutation analyses offered to MPS families by employing a dedicated member of staff to undertake these investigations.

Dr Robin Coffin £42,012: University College London Medical School, London, England
Final year of a two-year grant to research gene therapy for Mucopolysaccharide diseases using herpes simplex virus vectors.

Dr Bryan Winchester £44,000: Institute of Child Health, London, England
Final year of a four-year grant to establish mutational analysis in patients with Mucopolysaccharidoses types MPS I, MPS IIIA and MPS IIIB as a prerequisite for gene therapy and enzyme replacement therapy.

1998

Dr James Edmond Wraith £26,000: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
To fund 50% of year one of a three-year appointment for a second Consultant Paediatrician to specialise in the diagnosis and clinical management of MPS children and their families throughout the United Kingdom.

Dr Guy Besley £44,268: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
Year one of a three-year grant to extend the range of mutation analyses offered to MPS families by employing a dedicated member of staff to undertake these investigations.

Dr Robin Coffin £37,910: University College London Medical School, London, England
Year one of a two-year grant to research gene therapy for Mucopolysaccharide diseases using herpes simplex virus vectors.

Prof John Hopwood £15,000: Adelaide Women and Children’s Hospital, Adelaide, Australia
Final six months of an 18-month grant to develop high yielding expression systems to enable enzyme replacement therapy for MPS IVA, Morquio disease.

Dr Bryan Winchester £43,725: Institute of Child Health, London, England
Year three of a four-year grant to establish mutational analysis in patients with Mucopolysaccharidoses types MPS I, MPS IIIA and MPS IIIB as a prerequisite for gene therapy and enzyme replacement therapy.

1997

Dr Bryan Winchester £43,725: Institute of Child Health, London, England
Year two of a four-year grant to establish mutational analysis in patients with Mucopolysaccharidoses types MPS I, MPS IIIA and MPS IIIB as a prerequisite for gene therapy and enzyme replacement therapy.

Dr Linda Lashford £15,000: Patterson Institute, Christie Hospital, Manchester, England
50% of the final year of a two-year grant to optimise retro-viral vectors for gene delivery to the haemopoietic system in MPS I, Hurler disease.

Dr Bryan Winchester £1,200: Institute of Child Health, London, England
To look for mutations in Alpha Mannosidosis patients in collaboration with the Norwegian group at the University of Tromso who isolated the gene.

Dr James Edmond Wraith £41,880: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
Final year of a two-year grant working collaboratively with Dr Linda Lashford to support the clinical development of gene therapy by studying the efficacy of transplanted autologous retroviral transduced bone marrow in patients with MPSI, Hurler disease, and the development of gene transfer as a treatment strategy for patients with MPS II, Hunter disease.

Prof John Hopwood £30,000: Adelaide Women and Children’s Hospital, Adelaide, Australia
Year one of an 18-month grant to develop high yielding expression systems to enable enzyme replacement therapy for MPS IVA, Morquio disease

1996

Dr Charles Pennock £2,500: South Mead Health Services, Bristol, England
A grant to cover costs of reagents and consumables in identifying mutations of the iduronidase gene for MPS I.

Dr Bryan Winchester £5,000: Institute of Child Health, London, England
A grant to cover 5/8 for consumables for experimental gene therapy for Fucosidosis.

Dr Linda Lashford £16,295: Patterson Institute, Christie Hospital, Manchester, England
50% of the first year of a two-year grant to optimise retro-viral vectors for gene delivery to the haemopoietic system in MPS I, Hurler disease.

Dr Bryan Winchester £36,797: Institute of Child Health, London, England
Year one of a four-year grant to establish mutational analysis in patients with Mucopolysaccharidoses types MPS I, MPS IIIA and MPS IIIB as a prerequisite for gene therapy and enzyme replacement therapy.

Dr James Edmond Wraith £41,100: Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, England
Year one of a two-year grant working collaboratively with Dr Linda Lashford, to support the clinical development of gene therapy by studying the efficacy of transplanted autologous retroviral transduced bone marrow in patients with MPS I, Hurler disease, and the development of gene transfer as a treatment strategy for patients with MPS II, Hunter disease.