ハイデルベルグ大学
肝細胞移植
門脈内注射
CytoCur CytoNet
マルチ・センター(USA, Germany)
Synopsis
肝細胞移植
門脈内注射
CytoCur CytoNet
Durham-NC, USA
ほぐしたものを肝臓に入れている。マルチ・センター(USA, Germany)
SELICA III
スタディー・プロトコール SELICA IIPhase II/III
0.3 x 10^9 ６回に分けて入れる。
情報を手に入れなくてはいけない。Tx Control group vs
Synopsis
OCT deficiency: 3 cases
量は10^8細胞で十分なんだね。
OTC activity within the explanted liver 10 months after liver cell transplantation (LCT)
量は10^8細胞で十分なんだね。
OTC activity within the explanted liver 10 months after liver cell transplantation (LCT)
SSIEM SYMPOSIUM 2007
Liver cell transplantation for the treatment of inborn errors of metabolism
J. Meyburg & G. F. Hoffmann
Received: 1 December 2007 /Submitted in revised form: 1 February 2008 /Accepted: 5 February 2008 /Published online: 4 April 2008
# SSIEM and Springer 2008
Summary Over the last 15 years, liver cell transplantation
(LCT) has developed from an experimental laboratory
technique to a potentially life-saving therapeutic
option. Because of its minimally invasive nature, the
method is especially attractive for (small) children. In
children with liver-based inborn errors of metabolism,
this transfer of enzyme activity can be regarded as a
gene therapy, which can be installed independently and
additionally to conservative treatment concepts. To
date 14 children with inherited metabolic diseases have
undergone LCT in various centres. Although individual
results are encouraging, different treatment protocols,
difficulties in the objective assessment of function of the
transplant, and finally the lack of a controlled study
make it difficult to judge the overall significance of
LCT in the treatment of metabolic diseases and call
for collaborative clinical research.
Abbreviations
LCT liver cell transplantation
OLT orthotopic liver transplantation
Liver cell transplantation for the treatment of inborn errors of metabolism
J. Meyburg & G. F. Hoffmann
Received: 1 December 2007 /Submitted in revised form: 1 February 2008 /Accepted: 5 February 2008 /Published online: 4 April 2008
# SSIEM and Springer 2008
Summary Over the last 15 years, liver cell transplantation
(LCT) has developed from an experimental laboratory
technique to a potentially life-saving therapeutic
option. Because of its minimally invasive nature, the
method is especially attractive for (small) children. In
children with liver-based inborn errors of metabolism,
this transfer of enzyme activity can be regarded as a
gene therapy, which can be installed independently and
additionally to conservative treatment concepts. To
date 14 children with inherited metabolic diseases have
undergone LCT in various centres. Although individual
results are encouraging, different treatment protocols,
difficulties in the objective assessment of function of the
transplant, and finally the lack of a controlled study
make it difficult to judge the overall significance of
LCT in the treatment of metabolic diseases and call
for collaborative clinical research.
Abbreviations
LCT liver cell transplantation
OLT orthotopic liver transplantation
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