198 results on '"McIntosh, Jenny"'
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2. Transplacental delivery of therapeutic proteins by engineered immunoglobulin G: a step toward perinatal replacement therapy
3. Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome
4. Investigations of the thrombin generation test for the measurement of factor VIII
5. The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins
6. Liver Gene Therapy
7. 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.
8. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
9. Feasibility of combined upper and lower gastrointestinal endoscopic biopsy in the common marmoset (Callithrix jacchus) to evaluate gastrointestinal diseases
10. Feasibility of combined upper and lower gastrointestinal endoscopic biopsy in the common marmoset ( Callithrix jacchus ) to evaluate gastrointestinal diseases
11. Codon optimization of human factor VIII cDNAs leads to high-level expression
12. Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco
13. After the Melting Pot
14. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
15. Genetic architecture of tameness in a rat model of animal domestication
16. A robust non-human primate model of intrauterine gene therapy: Early- and late-gestation intervention with adeno-associated vectors: 7-1
17. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
18. Liver-Directed AAV Gene Therapy for Gaucher Disease
19. Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate
20. 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
21. Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera
22. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
23. The spectrum ofPIG-Agene mutations in aplastic anemia/paroxysmal nocturnal hemoglobinuria (AA/PNH): a high incidence of multiple mutations and evidence of a mutational hot spot
24. Liver directed AAV gene therapy to treat Gaucher disease
25. Liver-directed gene therapy corrects Fabry disease in mice
26. Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins
27. Therapeutic expression of human clotting factors IX and × following adeno‐associated viral vector‐mediated intrauterine gene transfer in early‐gestation fetal macaques
28. 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
29. Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy
30. A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX
31. 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
32. Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B
33. 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
34. Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
35. In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model
36. Hitzschlag – eine saisonale Herausforderung
37. Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.
38. Potential limits of AAV‐based gene therapy with the use of new transgenes expressing factor IX fusion proteins.
39. GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant
40. AAV-Mediated Expression of a Single-Chain FVIII Mimetic Antibody Efficiently Corrects Bleeding in Haemophilia a Mice
41. Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy
42. After the Melting Pot.
43. Die Auswirkungen Ketamin-basierter Narkoseprotokolle auf den intraokularen Druck bei der Katze – eine prospektive randomisierte Blindstudie: Die Auswirkungen Ketamin-basierter Narkoseprotokolle auf denintraokularen Druck bei der Katze– eine prospektive randomisierte Blindstudie
44. 171. Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus
45. Die Auswirkungen Ketamin-basierter Narkoseprotokolle auf den intraokularen Druck bei der Katze – eine prospektive randomisierte Blindstudie: Die Auswirkungen Ketamin-basierter Narkoseprotokolle auf denintraokularen Druck bei der Katze– eine prospektive randomisierte Blindstudie
46. Die Auswirkungen Ketamin-basierter Narkoseprotokolle auf den intraokularen Druck bei der Katze – eine prospektive randomisierte Blindstudie: Die Auswirkungen Ketamin-basierter Narkoseprotokolle auf denintraokularen Druck bei der Katze– eine prospektive randomisierte Blindstudie
47. Codon optimization of human factor VIII cDNAs leads to high-level expression
48. Genetic architecture of tameness in a rat model of animal domestication
49. Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B
50. AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage
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