Search Results - "Cullen, Karyn"
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Clinical, Electrophysiological, and Serum Biochemical Measures of Progressive Neurological and Hepatic Dysfunction in Feline Niemann-Pick Type C Disease
Published in Pediatric research (01-11-2008)“…Niemann-Pick type C (NP-C) disease is a neurovisceral lysosomal storage disease characterized by neurologic dysfunction, hepatosplenomegaly, and early death…”
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2
Mucopolysaccharidosis I Cats Mount a Cytotoxic T Lymphocyte Response after Neonatal Gene Therapy That Can Be Blocked with CTLA4-Ig
Published in Molecular therapy (01-07-2006)“…Although gene therapy has reduced manifestations of genetic diseases, immune responses can abrogate the effect. One approach to inducing tolerance is to…”
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3
Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice
Published in Thrombosis research (01-01-2007)“…Abstract Introduction Gene therapy could prevent bleeding in hemophilia. However, antibodies could inhibit coagulation, while cytotoxic T lymphocytes could…”
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Neonatal Gene Transfer Induces Tolerance to Human Factor VIII in Mice and Cats
Published in Blood (16-11-2005)“…Hemophilia A is a bleeding disorder due to Factor VIII (FVIII) deficiency. FVIII is a very immunogenic protein, as approximately 30% of patients develop…”
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859. Cats Produce a CTL Response That Reduces Expression of Human Factor IX and Human Factor VIII after Neonatal Gene Transfer and Represent an Important Model for Translation into Humans
Published in Molecular therapy (01-05-2006)“…Gene therapy has corrected bleeding manifestations of hemophilia in small and large animals. However, immune responses can abrogate an effect. We previously…”
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73. Cats Produce a Potent CTL Response after Neonatal Gene Therapy That Can Be Blocked with CTLA4-Ig
Published in Molecular therapy (01-05-2006)“…Although gene therapy has reduced manifestations of genetic diseases, immune responses can abrogate the effect. One approach to induce tolerance is to perform…”
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Journal Article