scholarly journals Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Types 2, 5, and 6 in Cystic Fibrosis and Normal Populations: Implications for Gene Therapy Using AAV Vectors

2006 ◽  
Vol 17 (4) ◽  
pp. 440-447 ◽  
Author(s):  
Christine L. Halbert ◽  
A. Dusty Miller ◽  
Sharon Mcnamara ◽  
Julia Emerson ◽  
Ronald L. Gibson ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Adeno Associated Virus ◽  
Normal Populations

Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Types 2, 5, and 6 in Cystic Fibrosis and Normal Populations: Implications for Gene Therapy Using AAV Vectors

2006 ◽  
Vol 0 (0) ◽  
pp. 060307123505001 ◽  
Author(s):  
Christine L. Halbert ◽  
A. Dusty Miller ◽  
Sharon McNamara ◽  
Julia Emerson ◽  
Ronald L. Gibson ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Adeno Associated Virus ◽  
Normal Populations

Adeno-associated virus as a gene therapy vector: strategies to neutralize the neutralizing antibodies

2019 ◽  
Vol 19 (3) ◽  
pp. 289-298 ◽  
Author(s):  
Majid Lotfinia ◽  
Meghdad Abdollahpour-Alitappeh ◽  
Behzad Hatami ◽  
Mohammad Reza Zali ◽  
Morteza Karimipoor
Keyword(s):  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Adeno Associated Virus ◽  
Gene Therapy Vector

Autoimmune anemia in macaques following erythropoietin gene therapy

Blood ◽  
2004 ◽  
Vol 103 (9) ◽  
pp. 3303-3304 ◽  
Author(s):  
Pierre Chenuaud ◽  
Thibaut Larcher ◽  
Joseph E. Rabinowitz ◽  
Nathalie Provost ◽  
Yan Cherel ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Gene Therapy ◽  
Autoimmune Disease ◽  
Neutralizing Antibodies ◽  
Adeno Associated Virus ◽  
Endogenous Erythropoietin ◽  
Cynomolgus Macaques ◽  
Vector Sequences ◽  
Gene Expressing ◽  
Self Antigen

Abstract We delivered the homologous erythropoietin (Epo) cDNA driven from a doxycycline-regulated promoter via recombinant adeno-associated virus in skeletal muscle of 9 cynomolgus macaques. Upon induction, rapid supraphysiologic levels of Epo were obtained. Unexpectedly, some individuals developed a profound anemia that correlated with the appearance of neutralizing antibodies against the endogenous Epo. Both the endogenous erythropoietin and vector sequences were identical. This is the first example of the inadvertent development of an autoimmune disease in primates as a result of gene transfer of a gene expressing a self-antigen. It raises some concerns when a therapeutic protein is produced at high levels from an ectopic site. (Blood. 2004;103:3303-3304)

scholarly journals Overcoming the Cystic Fibrosis Sputum Barrier to Leading Adeno-associated Virus Gene Therapy Vectors

2014 ◽  
Vol 22 (8) ◽  
pp. 1484-1493 ◽  
Author(s):  
Benjamin S Schuster ◽  
Anthony J Kim ◽  
Joshua C Kays ◽  
Mia M Kanzawa ◽  
William B Guggino ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Adeno Associated Virus ◽  
Virus Gene ◽  
Cystic Fibrosis Sputum ◽  
Gene Therapy Vectors
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