| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia
|
Graves, Lara E.
|
|
|
32 |
2 |
p.
|
artikel
|
| 2 |
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice
|
Sangster, Madison L.
|
|
|
32 |
2 |
p.
|
artikel
|
| 3 |
Adeno-associated virus perfusion enhanced expression: A commercially scalable, high titer, high quality producer cell line process
|
Xue, Wei
|
|
|
32 |
2 |
p.
|
artikel
|
| 4 |
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development
|
Schimek, Adrian
|
|
|
32 |
2 |
p.
|
artikel
|
| 5 |
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients
|
Liu, Li
|
|
|
32 |
2 |
p.
|
artikel
|
| 6 |
A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease
|
Hart, Kevyn L.
|
|
|
32 |
2 |
p.
|
artikel
|
| 7 |
A robust and flexible baculovirus-insect cell system for AAV vector production with improved yield, capsid ratios and potency
|
Marwidi, Yoko
|
|
|
32 |
2 |
p.
|
artikel
|
| 8 |
A toxicology study of Csf2ra complementation and pulmonary macrophage transplantation therapy of hereditary PAP in mice
|
Arumugam, Paritha
|
|
|
32 |
2 |
p.
|
artikel
|
| 9 |
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy
|
Yonezawa Ogusuku, Isabella Elias
|
|
|
32 |
2 |
p.
|
artikel
|
| 10 |
Bioengineering extracellular vesicle cargo for optimal therapeutic efficiency
|
René, Charlotte A.
|
|
|
32 |
2 |
p.
|
artikel
|
| 11 |
Biomanufacturing in gene and cell therapy
|
Stone, Daniel
|
|
|
32 |
2 |
p.
|
artikel
|
| 12 |
CAR-T cell manufacturing landscape—Lessons from the past decade and considerations for early clinical development
|
Dias, Juliana
|
|
|
32 |
2 |
p.
|
artikel
|
| 13 |
Combining CRISPR-Cas-mediated terminal resolution with a novel genetic workflow to achieve high-diversity adenoviral libraries
|
Fischer, Julian
|
|
|
32 |
2 |
p.
|
artikel
|
| 14 |
Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy
|
Ellison, Stuart
|
|
|
32 |
2 |
p.
|
artikel
|
| 15 |
Development and testing of a versatile genome editing application reporter (V-GEAR) system
|
Kleinboehl, Evan W.
|
|
|
32 |
2 |
p.
|
artikel
|
| 16 |
Development of adenoviral vectors that transduce Purkinje cells and other cerebellar cell-types in the cerebellum of a humanized mouse model
|
Kul, Emre
|
|
|
32 |
2 |
p.
|
artikel
|
| 17 |
Development of novel lipoplex formulation methodologies to improve large-scale transient transfection for lentiviral vector manufacture
|
Williams-Fegredo, Thomas
|
|
|
32 |
2 |
p.
|
artikel
|
| 18 |
Disseminating transformative gene and cell therapy research
|
Abou-el-Enein, Mohamed
|
|
|
32 |
2 |
p.
|
artikel
|
| 19 |
Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy
|
Audouard, Emilie
|
|
|
32 |
2 |
p.
|
artikel
|
| 20 |
Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs
|
Tang, Yinghua
|
|
|
32 |
2 |
p.
|
artikel
|
| 21 |
Embryo and fetal gene editing: Technical challenges and progress toward clinical applications
|
Mattar, Citra N.Z.
|
|
|
32 |
2 |
p.
|
artikel
|
| 22 |
Endovascular transplantation of mRNA-enhanced mesenchymal stromal cells results in superior therapeutic protein expression in swine heart
|
Al-Saadi, Jonathan
|
|
|
32 |
2 |
p.
|
artikel
|
| 23 |
Epigenetic control of multiple genes with a lentiviral vector encoding transcriptional repressors fused to compact zinc finger arrays
|
Monteferrario, Davide
|
|
|
32 |
2 |
p.
|
artikel
|
| 24 |
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2
|
Prabhakar, Shilpa
|
|
|
32 |
2 |
p.
|
artikel
|
| 25 |
Genetic alteration of SJ293TS cells and modification of serum-free media enhances lentiviral vector production
|
Bauler, Matthew
|
|
|
32 |
2 |
p.
|
artikel
|
| 26 |
Glycosylation of recombinant adeno-associated virus serotype 6
|
Yamaguchi, Yuki
|
|
|
32 |
2 |
p.
|
artikel
|
| 27 |
Identifying MAGE-A4-positive tumors for TCR T cell therapies in HLA-A∗02-eligible patients
|
Wang, Tianjiao
|
|
|
32 |
2 |
p.
|
artikel
|
| 28 |
It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome
|
Naseem, Asma
|
|
|
32 |
2 |
p.
|
artikel
|
| 29 |
Manufacturing DNA in E. coli yields higher-fidelity DNA than in vitro enzymatic synthesis
|
Hersch, Steven J.
|
|
|
32 |
2 |
p.
|
artikel
|
| 30 |
Metabolic priming of GD2 TRAC-CAR T cells during manufacturing promotes memory phenotypes while enhancing persistence
|
Cappabianca, Dan
|
|
|
32 |
2 |
p.
|
artikel
|
| 31 |
mtDNA analysis using Mitopore
|
Dobner, Jochen
|
|
|
32 |
2 |
p.
|
artikel
|
| 32 |
Nanodysferlins support membrane repair and binding to TRIM72/MG53 but do not localize to t-tubules or stabilize Ca2+ signaling
|
Muriel, Joaquin
|
|
|
32 |
2 |
p.
|
artikel
|
| 33 |
Novel AAV variants with improved tropism for human Schwann cells
|
Drouyer, Matthieu
|
|
|
32 |
2 |
p.
|
artikel
|
| 34 |
Peptide-encoding gene transfer to modulate intracellular protein-protein interactions
|
Taya, Toshihiko
|
|
|
32 |
2 |
p.
|
artikel
|
| 35 |
Preclinical specificity & activity of a fully human 41BB-expressing anti-CD19 CART- therapy for treatment-resistant autoimmune disease
|
Peng, Binghao J.
|
|
|
32 |
2 |
p.
|
artikel
|
| 36 |
Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus
|
Borges, Beltran
|
|
|
32 |
2 |
p.
|
artikel
|
| 37 |
Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection
|
Liu, Hao
|
|
|
32 |
2 |
p.
|
artikel
|
| 38 |
Promise of gene therapy for congenital neurologic disease due to GPI deficiency
|
Babushok, Daria V.
|
|
|
32 |
2 |
p.
|
artikel
|
| 39 |
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy
|
Li, Xueyi
|
|
|
32 |
2 |
p.
|
artikel
|
| 40 |
Quasi-perfusion studies for intensified lentiviral vector production using a continuous stable producer cell line
|
Stibbs, Dale J.
|
|
|
32 |
2 |
p.
|
artikel
|
| 41 |
Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency
|
Fernandes-Pires, Gabriella
|
|
|
32 |
2 |
p.
|
artikel
|
| 42 |
SERCA2a overexpression improves muscle function in a canine Duchenne muscular dystrophy model
|
Kodippili, Kasun
|
|
|
32 |
2 |
p.
|
artikel
|
| 43 |
siRNA-mediated reduction of a circulating protein in swine using lipid nanoparticles
|
Cau, Massimo F.
|
|
|
32 |
2 |
p.
|
artikel
|
| 44 |
Strengthening health systems for access to gene therapy in rare genetic disorders
|
Bhatia, Sonal
|
|
|
32 |
2 |
p.
|
artikel
|
| 45 |
Subretinal AAV delivery of RNAi-therapeutics targeting VEGFA reduces choroidal neovascularization in a large animal model
|
Haldrup, Silja Hansen
|
|
|
32 |
2 |
p.
|
artikel
|
| 46 |
Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection
|
Hunter, Jacqueline E.
|
|
|
32 |
2 |
p.
|
artikel
|
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