scholarly journals High efficiency myogenic conversion of human fibroblasts by adenoviral vector-mediated MyoD gene transfer. An alternative strategy for ex vivo gene therapy of primary myopathies.

1998 ◽  
Vol 101 (10) ◽  
pp. 2119-2128 ◽  
Author(s):  
L Lattanzi ◽  
G Salvatori ◽  
M Coletta ◽  
C Sonnino ◽  
M G Cusella De Angelis ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Adenoviral Vector ◽  
High Efficiency ◽  
Ex Vivo ◽  
Human Fibroblasts ◽  
Alternative Strategy ◽  
Ex Vivo Gene Therapy ◽  
Myod Gene

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)–deficient mice and corrects their immune and metabolic defects

Blood ◽  
2006 ◽  
Vol 108 (9) ◽  
pp. 2979-2988 ◽  
Author(s):  
Alessandra Mortellaro ◽  
Raisa Jofra Hernandez ◽  
Matteo M. Guerrini ◽  
Filippo Carlucci ◽  
Antonella Tabucchi ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Bone Marrow ◽  
Gene Transfer ◽  
Adenosine Deaminase ◽  
Ex Vivo ◽  
Severe Combined Immunodeficiency ◽  
Lentiviral Vectors ◽  
Mixed Chimerism ◽  
Pulmonary Insufficiency ◽  
Ex Vivo Gene Therapy

AbstractAdenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe combined immunodeficiency (SCID) and multiple organ damage. To investigate the efficacy of ex vivo gene therapy with self-inactivating lentiviral vectors (LVs) in correcting this complex phenotype, we used an ADA–/– mouse model characterized by early postnatal lethality. LV-mediated ADA gene transfer into bone marrow cells combined with low-dose irradiation rescued mice from lethality and restored their growth, as did transplantation of wild-type bone marrow. Mixed chimerism with multilineage engraftment of transduced cells was detected in the long term in animals that underwent transplantation. ADA activity was normalized in lymphocytes and partially corrected in red blood cells (RBCs), resulting in full metabolic detoxification and prevention of severe pulmonary insufficiency. Moreover, gene therapy restored normal lymphoid differentiation and immune functions, including antigen-specific antibody production. Similar degrees of detoxification and immune reconstitution were obtained in mice treated early after birth or after 1 month of enzyme-replacement therapy, mimicking 2 potential applications for ADA-SCID. Overall, this study demonstrates the efficacy of LV gene transfer in correcting both the immunological and metabolic phenotypes of ADA-SCID and supports the future clinical use of this approach.

scholarly journals The Mechanical Agitation Method of Gene Transfer for Ex-Vivo Gene Therapy

10.5772/21477 ◽  
2011 ◽  
Author(s):  
Hea-Jong Chung ◽  
Hyun-Seo Lee ◽  
Hyeon-Jin Kim ◽  
Seong-Tshool Hong
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Ex Vivo ◽  
Mechanical Agitation ◽  
Ex Vivo Gene Therapy

scholarly journals Ex-Vivo Gene Therapy Using Lentiviral Mediated Gene Transfer Into Umbilical Cord Blood Derived Stem Cells

2016 ◽  
Vol In Press (InPress) ◽  
Author(s):  
Hanieh Jalali ◽  
Kazem Parivar ◽  
Masoud Soleimani ◽  
Mohammad Nabiuni ◽  
Hamid Aghaee-Bakhtiari
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Gene Transfer ◽  
Cord Blood ◽  
Umbilical Cord ◽  
Umbilical Cord Blood ◽  
Ex Vivo ◽  
Ex Vivo Gene Therapy

Overcoming the Immune Response to Permit Ex Vivo Gene Therapy for Spine Fusion With Human Type 5 Adenoviral Delivery of the LIM Mineralization Protein-1 cDNA

Spine ◽  
2003 ◽  
Vol 28 (3) ◽  
pp. 219-226 ◽  
Author(s):  
Hak-Sun Kim ◽  
Manjula Viggeswarapu ◽  
Scott D. Boden ◽  
Yunshan Liu ◽  
Gregory A Hair ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Immune Response ◽  
Ex Vivo ◽  
Spine Fusion ◽  
Human Type ◽  
Adenoviral Delivery ◽  
Ex Vivo Gene Therapy
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