515 LENTIVIRAL ‘GENE ONTOLOGY’ (LEGO) VECTORS ENABLE HIGHLY EFFICIENT EX VIVO TRANSDUCTION OF HEPATOCYTES SUITABLE FOR CELL TRANSPLANTATION

2011 ◽  
Vol 54 ◽  
pp. S211-S212
Author(s):  
M. Warlich ◽  
K. Weber ◽  
M. Thomaschewski ◽  
M. Lütgehetmann ◽  
T. Volz ◽  
...  
Blood ◽  
2010 ◽  
Vol 116 (26) ◽  
pp. 6123-6132 ◽  
Author(s):  
Richard K. Burt ◽  
Robert M. Craig ◽  
Francesca Milanetti ◽  
Kathleen Quigley ◽  
Paula Gozdziak ◽  
...  

Abstract We evaluated the safety and clinical outcome of autologous nonmyeloablative hematopoietic stem cell transplantation (HSCT) in patients with severe Crohn disease (CD) defined as a Crohn Disease Activity Index (CDAI) greater than 250, and/or Crohn Severity Index greater than 16 despite anti–tumor necrosis factor therapy. Stem cells were mobilized from the peripheral blood using cyclophosphamide (2.0 g/m2) and G-CSF (10 μg/kg/day), enriched ex vivo by CD34+ selection, and reinfused after immune suppressive conditioning with cyclophosphamide (200 mg/kg) and either equine antithymocyte globulin (ATG, 90 mg/kg) or rabbit ATG (6 mg/kg). Eighteen of 24 patients are 5 or more years after transplantation. All patients went into remission with a CDAI less than 150. The percentage of clinical relapse-free survival defined as the percent free of restarting CD medical therapy after transplantation is 91% at 1 year, 63% at 2 years, 57% at 3 years, 39% at 4 years, and 19% at 5 years. The percentage of patients in remission (CDAI < 150), steroid-free, or medication-free at any posttransplantation evaluation interval more than 5 years after transplantation has remained at or greater than 70%, 80%, and 60%, respectively. This trial was registered at www.clinicaltrials.gov as NCT0027853.


Circulation ◽  
2001 ◽  
Vol 104 (suppl_1) ◽  
Author(s):  
Bruno Pouzet ◽  
Saïd Ghostine ◽  
Jean-Thomas Vilquin ◽  
Isabelle Garcin ◽  
Marcio Scorsin ◽  
...  

Background There is compelling experimental evidence that autologous skeletal muscle (SM) cell transplantation improves postinfarction cardiac function. This study assessed whether this benefit is still manifested in the clinically relevant setting of a treatment by ACE inhibitors. Methods and Results A myocardial infarction was created in 99 rats by coronary artery ligation. They were divided into 4 groups. Two groups did not receive any drug and were intramyocardially injected 7 days after the infarct with either culture medium alone (control rats, n=16) or autologous SM cells (2.3×10 6 myoblasts) previously expanded ex vivo for 7 days (myoblasts, n=24). Two other groups received the ACE inhibitor perindoprilat (1 mg · kg −1 · d −1 ), started the day of the infarct and continued uninterruptedly thereafter, and underwent time-matched procedures, that is, they were intramyocardially injected at 7 days after infarction with either culture medium alone (ACE inhibitors, n=22) or autologous SM cells (2.5×10 6 myoblasts) previously expanded ex vivo for 7 days (ACE inhibitors+myoblasts, n=37). Left ventricular function was assessed by 2D echocardiography. At the end of the 2-month study, left ventricular ejection fraction (%, mean±SEM) was increased in all groups (myoblasts, 37.4±1.2; ACE inhibitors, 31.6±1.7; ACE inhibitors+myoblasts, 43.9±1.4) compared with that in control rats (19.8±0.7) ( P <0.0001). The improvement in ejection fraction was similar in the ACE inhibitor and the myoblast groups (31.6±1.7 versus 37.4±1.2, P =0.0636). However, in the ACE inhibitor+myoblast group, this improvement was greater than that seen in hearts receiving either treatment alone (43.9±1.4 versus 31.6±1.7 in the ACE inhibitor group and 43.9±1.4. versus 37.4±1.2 in the myoblast group, P <0.0001 and P =0.0084, respectively). Conclusions These data provide further support for the clinical relevance of autologous SM cell transplantation in that its cardioprotective effects are additive to those observed with ACE inhibitors.


2015 ◽  
Vol 1 ◽  
pp. 19
Author(s):  
L.E. Hogan ◽  
K.S. Hobbs ◽  
D.R. Kuritzkes ◽  
J. Ritz ◽  
T.J. Henrich

Biomedicines ◽  
2020 ◽  
Vol 8 (6) ◽  
pp. 157
Author(s):  
Nicole Zarniko ◽  
Anna Skorska ◽  
Gustav Steinhoff ◽  
Robert David ◽  
Ralf Gaebel

Several cell populations derived from bone marrow (BM) have been shown to possess cardiac regenerative potential. Among these are freshly isolated CD133+ hematopoietic as well as culture-expanded mesenchymal stem cells. Alternatively, by purifying CD271+ cells from BM, mesenchymal progenitors can be enriched without an ex vivo cultivation. With regard to the limited available number of freshly isolated BM-derived stem cells, the effect of the dosage on the therapeutic efficiency is of particular interest. Therefore, in the present pre-clinical study, we investigated human BM-derived CD133+ and CD271+ stem cells for their cardiac regenerative potential three weeks post-myocardial infarction (MI) in a dose-dependent manner. The improvement of the hemodynamic function as well as cardiac remodeling showed no therapeutic difference after the transplantation of both 100,000 and 500,000 stem cells. Therefore, beneficial stem cell transplantation post-MI is widely independent of the cell dose and detrimental stem cell amplification in vitro can likely be avoided.


2016 ◽  
Vol 17 (4) ◽  
pp. 721-734 ◽  
Author(s):  
Wichaya Sriuttha ◽  
Nantawat Uttamo ◽  
Apisek Kongkaew ◽  
Jongkolnee Settakorn ◽  
Suchanan Rattanasalee ◽  
...  

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