Gene therapy: Lessons learned from liver transplantation for transthyretin-amyloidosis

Liver Transplantation

Volumn 10, Issue 12, 2004, Pages 1551-1553

  Suhr, Ole B ^a   Holmgren, Gösta ^a   Lundgren, Erik ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

AMYLOID; ATELOCOLLAGEN; DNA; PREALBUMIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL BASED GENE THERAPY; CELL STRAIN HEPG2; DNA REPAIR; FAMILIAL AMYLOID POLYNEUROPATHY; GENE AMPLIFICATION; GENE CONVERSION; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; LIVER; LIVER TRANSPLANTATION; MOUSE; NONHUMAN; PERIPHERAL NERVE; PRIORITY JOURNAL; PROTEIN SYNTHESIS; REAL TIME POLYMERASE CHAIN REACTION; TRANSGENIC MOUSE; WILD TYPE;

AMYLOID NEUROPATHIES, FAMILIAL; GENE THERAPY; HUMANS; LIVER TRANSPLANTATION; PREALBUMIN; VARIATION (GENETICS);

EID: 11144255024     PISSN: 15276465     EISSN: None     Source Type: Journal    
DOI: 10.1002/lt.20302     Document Type: Article

References (9)

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2. Nakamura M, Ando Y, Nagahara S, Sano A, Ochiya T, Maeda S, et al. Targeted conversion of the transthyretin gene in vitro and in vivo. Gene Ther 2004;11:838-846.
3. Nathwani AC, Benjamin R, Nienhuis AW, Davidoff AM. Current status and prospects for gene therapy. Vox Sang 2004;87:73-81.
4. Holmgren G, Steen L, Ekstedt J, Groth CG, Ericzon BG, Eriksson S, et al. Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30). Clin Genet 1991;40:242-246.
5. Suhr OB, Ericzon BG, Friman S. Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type). Liver Transpl 2002;8:787-794.
6. Westermark P, Sletten K, Johansson B, Cornwell GGd. Fibril in senile systemic amyloidosis is derived from normal transthyretin. Proc Natl Acad Sci USA 1990;87:2843-2845.
7. Hornsten R, Wiklund U, Olofsson BO, Jensen SM, Suhr OB. Liver transplantation does not prevent the development of life-threatening arrhythmia in familial amyloidotic polyneuropathy, Portuguese-type (ATTR Val30Met) patients. Transplantation 2004;78:112-116.
8. Sharma P, Perri RE, Sirven JE, Zeldenrust SR, Brandhagen DJ, Rosen CB, et al. Outcome of liver transplantation for familial amyloidotic polyneuropathy. Liver Transpl 2003;9:1273-1280.
9. Yazaki M, Tokuda T, Nakamura A, Higashikata T, Koyama J, Higuchi K, et al. Cardiac amyloid in patients with familial amyloid polyneuropathy consists of abundant wild-type transthyretin. Biochem Biophys Res Commun 2000;274:702-706.