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Review 1. Enzyme-replacement therapy: problems and prospects.

Authors: B Rademaker; J Raber
Journal: Pharm Weekbl Sci Date: 1989-10-20

2. Female with Fabry Disease Unknowingly Donates Affected Kidney to Sister: A Call for Pre-transplant Genetic Testing.

Authors: Lindsay S Paull; Michael J Lipinski; William G Wilson; Shawn E Lipinski
Journal: JIMD Rep Date: 2011-12-06

3. Alternative strategies for gene therapy of hemophilia.

Authors: Robert R Montgomery; Qizhen Shi
Journal: Hematology Am Soc Hematol Educ Program Date: 2010

Review 4. Lysosomal storage diseases: mechanisms of enzyme replacement therapy.

Authors: G Bou-Gharios; D Abraham; I Olsen
Journal: Histochem J Date: 1993-09

5. Long-term effects of bone marrow transplantation on lysosomal enzyme replacement in beta-glucuronidase-deficient mice.

Authors: G Bou-Gharios; G Adams; P Pace; I Olsen
Journal: J Inherit Metab Dis Date: 1992 Impact factor: 4.982

6. The development of anti-glomerular basement membrane nephritis in two children with Alport's syndrome after renal transplantation: characterization of the antibody target.

Authors: L P vd Heuvel; C H Schröder; C O Savage; D Menzel; K J Assmann; L A Monnens; J H Veerkamp
Journal: Pediatr Nephrol Date: 1989-10 Impact factor: 3.714

7. A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.

Authors: Scot A Fahs; Matthew T Hille; Qizhen Shi; Hartmut Weiler; Robert R Montgomery
Journal: Blood Date: 2014-04-04 Impact factor: 22.113

Review 8. Treatment of inherited metabolic disorders by liver transplantation.

Authors: M Burdelski; B Rodeck; A Latta; K Latta; J Brodehl; B Ringe; R Pichlmayr
Journal: J Inherit Metab Dis Date: 1991 Impact factor: 4.982

9. Mesenchymal Stromal Cells in Pediatric Hematopoietic Cell Transplantation a Review and a Pilot Study in Children Treated With Decidua Stromal Cells for Acute Graft-versus-Host Disease.

Authors: Olle Ringdén; Britt Gustafsson; Behnam Sadeghi
Journal: Front Immunol Date: 2020-10-19 Impact factor: 7.561

9 in total