51 results on '"McIntosh, Jenny"'
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2. Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome
3. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
4. Liver Gene Therapy
5. The use of a transient transfected expression system to deliver high quality bispecific T-cell engager drug product, NVG-111, to the clinic for a fraction of the cost and time associated with the development and use of a producer cell line.
6. Feasibility of combined upper and lower gastrointestinal endoscopic biopsy in the common marmoset ( Callithrix jacchus ) to evaluate gastrointestinal diseases
7. Liver-Directed AAV Gene Therapy for Gaucher Disease
8. Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate
9. Liver directed AAV gene therapy to treat Gaucher disease
10. Liver-directed gene therapy corrects Fabry disease in mice
11. Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins
12. Therapeutic expression of human clotting factors IX and × following adeno‐associated viral vector‐mediated intrauterine gene transfer in early‐gestation fetal macaques
13. A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B
14. Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy
15. A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX
16. GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant
17. Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B
18. Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer
19. Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
20. The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins
21. In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model
22. Hitzschlag – eine saisonale Herausforderung
23. 171. Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus
24. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
25. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
26. Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B
27. AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage
28. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
29. Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B
30. Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques
31. Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial
32. Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins
33. Recombinant Adeno-Associated Virus-Mediated In Utero Gene Transfer Gives Therapeutic Transgene Expression in the Sheep
34. Codon optimization of human factor VIII cDNAs leads to high-level expression
35. Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B
36. Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System
37. Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant
38. Genetic Architecture of Tameness in a Rat Model of Animal Domestication
39. Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice
40. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors
41. Luciferin Detection After Intranasal Vector Delivery Is Improved by Intranasal Rather Than Intraperitoneal Luciferin Administration
42. Luciferin detection after intra-nasal vector delivery is improved by intra-nasal rather than intra-peritoneal luciferin administration.
43. Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats
44. Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera
45. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
46. Continuous delivery of human type I interferons (α/β) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model
47. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
48. Comparison of Viral Vectors for Gene Transfer into CLL Cells: Efficient Transduction with Adeno-Associated Virus-8 (AAV-8).
49. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
50. The spectrum of PIG-A gene mutations in aplastic anemia/paroxysmal nocturnal hemoglobinuria (AA/PNH): a high incidence of multiple mutations and evidence of a mutational hot spot
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