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1. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

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

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

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

24. Liver directed AAV gene therapy to treat Gaucher disease

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

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

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

35. In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

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

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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