| scholarly article | Q13442814 |
| P2093 | author name string | Katherine A High | |
| P2860 | cites work | Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial | Q46415131 |
| Functional and structural evaluation after AAV.RPE65 gene transfer in the canine model of Leber's congenital amaurosis | Q47389112 | ||
| Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver | Q48004746 | ||
| Lessons from TGN1412. | Q55042674 | ||
| Natural history of HCV infection | Q57782866 | ||
| The canine factor VIII cDNA and 5' flanking sequence | Q74286665 | ||
| Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus | Q77292224 | ||
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
| Highly efficient endogenous human gene correction using designed zinc-finger nucleases | Q28243157 | ||
| Gene targeting using zinc finger nucleases | Q28265983 | ||
| Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
| Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases | Q29615069 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Cytokine storm in a phase 1 trial of the anti-CD28 monoclonal antibody TGN1412 | Q29622893 | ||
| The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion | Q30745198 | ||
| Congenital stationary night blindness in the dog: common mutation in the RPE65 gene indicates founder effect. | Q31978440 | ||
| Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer | Q33315634 | ||
| Retinal dystrophy of Swedish briard/briard-beagle dogs is due to a 4-bp deletion in RPE65. | Q33857732 | ||
| PTC124 targets genetic disorders caused by nonsense mutations | Q34003720 | ||
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 | ||
| Canine hemophilia B resulting from a point mutation with unusual consequences | Q34326780 | ||
| Loss of Siglec expression on T lymphocytes during human evolution | Q34650845 | ||
| CD28 superagonists: what makes the difference in humans? | Q34656318 | ||
| Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer | Q34985105 | ||
| Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency | Q35006603 | ||
| Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector | Q35872651 | ||
| Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein | Q35943644 | ||
| Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. | Q36007714 | ||
| Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus | Q36159178 | ||
| Quantifying the federal minimal risk standard: implications for pediatric research without a prospect of direct benefit. | Q36232638 | ||
| Treatment of human disease by adeno-associated viral gene transfer | Q36448393 | ||
| The Jeremiah Metzger lecture. Current status of human gene therapy. | Q36646925 | ||
| Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness | Q36825024 | ||
| Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics | Q36937189 | ||
| Immune responses to AAV in clinical trials | Q36990095 | ||
| Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy | Q37071092 | ||
| Manufacturing and characterizing AAV-based vectors for use in clinical studies | Q37140657 | ||
| Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses | Q39501488 | ||
| Haemophilia prophylaxis in young patients--a long-term follow-up | Q41497194 | ||
| Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. | Q41722852 | ||
| Gene therapy restores vision in a canine model of childhood blindness | Q43590733 | ||
| De novo use of sirolimus in immunosuppression regimens in kidney and kidney-pancreas transplantation at the University of California, San Francisco | Q44437985 | ||
| De novo kidney transplantation without use of calcineurin inhibitors preserves renal structure and function at two years. | Q45100299 | ||
| Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors | Q45299117 | ||
| CD8(+) T-cell responses to adeno-associated virus capsid in humans | Q45406111 | ||
| Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography | Q45408693 | ||
| Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo | Q45709105 | ||
| Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector | Q45855552 | ||
| A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency | Q45857796 | ||
| Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector | Q45862203 | ||
| T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. | Q45866838 | ||
| Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A | Q45868121 | ||
| Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation | Q45879591 | ||
| Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males | Q45881920 | ||
| Gene therapy: the moving finger | Q45883940 | ||
| A coagulation factor IX-deficient mouse model for human hemophilia B. | Q45884173 | ||
| Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy | Q45884700 | ||
| Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy | Q45888356 | ||
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 331-359 | |
| P577 | publication date | 2009-01-01 | |
| P1433 | published in | Transactions of the American Clinical and Climatological Association | Q24056174 |
| P1476 | title | The Jeremiah Metzger Lecture: gene therapy for inherited disorders: from Christmas disease to Leber's amaurosis | |
| P478 | volume | 120 |
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