| no |
title |
author |
magazine |
year |
volume |
issue |
page(s) |
type |
| 1 |
AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver
|
Greig, Jenny A.
|
|
2018
|
11 |
C |
p. 191-201
|
article
|
| 2 |
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer
|
Wang, Dan
|
|
2018
|
11 |
C |
p. 65-72
|
article
|
| 3 |
Alpha 1 Antitrypsin Gene Therapy Extends the Lifespan of Lupus-Prone Mice
|
Elshikha, Ahmed Samir
|
|
2018
|
11 |
C |
p. 131-142
|
article
|
| 4 |
Barcoding of Macaque Hematopoietic Stem and Progenitor Cells: A Robust Platform to Assess Vector Genotoxicity
|
Yabe, Idalia M.
|
|
2018
|
11 |
C |
p. 143-154
|
article
|
| 5 |
Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe
|
ten Ham, Renske M.T.
|
|
2018
|
11 |
C |
p. 121-130
|
article
|
| 6 |
Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain
|
Quintino, Luis
|
|
2018
|
11 |
C |
p. 29-39
|
article
|
| 7 |
Development of a Novel Recombinant Adeno-Associated Virus Production System Using Human Bocavirus 1 Helper Genes
|
Wang, Zekun
|
|
2018
|
11 |
C |
p. 40-51
|
article
|
| 8 |
Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice
|
Vannoy, Charles Harvey
|
|
2018
|
11 |
C |
p. 106-120
|
article
|
| 9 |
Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome
|
Han, Jing-fen
|
|
2018
|
11 |
C |
p. 52-64
|
article
|
| 10 |
Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9
|
Tomono, Taro
|
|
2018
|
11 |
C |
p. 180-190
|
article
|
| 11 |
IPRF Reflection Paper on Biodistribution
|
Havert, Mike
|
|
2018
|
11 |
C |
p. 166
|
article
|
| 12 |
Lentiviral Vector Purification Using Genetically Encoded Biotin Mimic in Packaging Cell
|
Mekkaoui, Leila
|
|
2018
|
11 |
C |
p. 155-165
|
article
|
| 13 |
MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7
|
Luo, Xufeng
|
|
2018
|
11 |
C |
p. 73-82
|
article
|
| 14 |
Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice
|
Kennedy, Tahnee L.
|
|
2018
|
11 |
C |
p. 92-105
|
article
|
| 15 |
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
|
Lidonnici, Maria Rosa
|
|
2018
|
11 |
C |
p. 9-28
|
article
|
| 16 |
P-FN12, an H4R-Based Epitope Vaccine Screened by Phage Display, Regulates the Th1/Th2 Balance in Rat Allergic Rhinitis
|
Wang, Yuqian
|
|
2018
|
11 |
C |
p. 83-91
|
article
|
| 17 |
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
|
Poletti, Valentina
|
|
2018
|
11 |
C |
p. 167-179
|
article
|
| 18 |
Validation of an Immunoassay for Anti-thymidine Phosphorylase Antibodies in Patients with MNGIE Treated with Enzyme Replacement Therapy
|
Levene, Michelle
|
|
2018
|
11 |
C |
p. 1-8
|
article
|
Journal description