| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-mediated BMP7 gene therapy counteracts insulin resistance and obesity
|
Casana, Estefania
|
|
|
25 |
C |
p. 190-204
|
artikel
|
| 2 |
AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice
|
Palfi, Arpad
|
|
|
25 |
C |
p. 236-249
|
artikel
|
| 3 |
A chimeric virus-based probe unambiguously detects live circulating tumor cells with high specificity and sensitivity
|
Fu, Xinping
|
|
|
25 |
C |
p. 136
|
artikel
|
| 4 |
A combinatorial CRISPR-Cas12a attack on HIV DNA
|
Fan, Minghui
|
|
|
25 |
C |
p. 43-51
|
artikel
|
| 5 |
A flexible, thermostable nanostructured lipid carrier platform for RNA vaccine delivery
|
Gerhardt, Alana
|
|
|
25 |
C |
p. 205-214
|
artikel
|
| 6 |
An etanercept O-glycovariant with enhanced potency
|
Biel, Thomas G.
|
|
|
25 |
C |
p. 124-135
|
artikel
|
| 7 |
A repositioning screen using an FGFR2 splicing reporter reveals compounds that regulate epithelial-mesenchymal transitions and inhibit growth of prostate cancer xenografts
|
Li, Ling
|
|
|
25 |
C |
p. 147-157
|
artikel
|
| 8 |
A systematic comparison of optogenetic approaches to visual restoration
|
Gilhooley, Michael J.
|
|
|
25 |
C |
p. 111-123
|
artikel
|
| 9 |
Biodistribution and environmental safety of a live-attenuated YF17D-vectored SARS-CoV-2 vaccine candidate
|
Li, Li-Hsin
|
|
|
25 |
C |
p. 215-224
|
artikel
|
| 10 |
Characterizing piggyBat—a transposase for genetic modification of T cells
|
Sutrave, Gaurav
|
|
|
25 |
C |
p. 250-263
|
artikel
|
| 11 |
Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species
|
Fader, Kelly A.
|
|
|
25 |
C |
p. 264-277
|
artikel
|
| 12 |
Combined overexpression of ATXN1L and mutant ATXN1 knockdown by AAV rescue motor phenotypes and gene signatures in SCA1 mice
|
Carrell, Ellie M.
|
|
|
25 |
C |
p. 333-343
|
artikel
|
| 13 |
Correction of a chronic pulmonary disease through lentiviral vector-mediated protein expression
|
Lund-Palau, Helena
|
|
|
25 |
C |
p. 382-391
|
artikel
|
| 14 |
Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice
|
Morimoto, Hideto
|
|
|
25 |
C |
p. 534-544
|
artikel
|
| 15 |
Editing T cell repertoire by thymic epithelial cell-directed gene transfer abrogates risk of type 1 diabetes development
|
Russo, Fabio
|
|
|
25 |
C |
p. 508-519
|
artikel
|
| 16 |
Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine
|
Bianchi, Marzia
|
|
|
25 |
C |
p. 26-40
|
artikel
|
| 17 |
Follistatin-like 1 promotes proliferation of matured human hypoxic iPSC-cardiomyocytes and is secreted by cardiac fibroblasts
|
Peters, Marijn C.
|
|
|
25 |
C |
p. 3-16
|
artikel
|
| 18 |
Gene therapy for Friedreich ataxia: Too much, too little, or just right?
|
Payne, R. Mark
|
|
|
25 |
C |
p. 1-2
|
artikel
|
| 19 |
Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities
|
Khoja, Suhail
|
|
|
25 |
C |
p. 278-296
|
artikel
|
| 20 |
Good reasons for targeting SARS-CoV-2 by engineered extracellular vesicles
|
de Marco, Ario
|
|
|
25 |
C |
p. 41-42
|
artikel
|
| 21 |
Haplotype-specific insertion-deletion variations for allele-specific targeting in Huntington's disease
|
Shin, Jun Wan
|
|
|
25 |
C |
p. 84-95
|
artikel
|
| 22 |
Hematopoietic stem cell gene therapy ameliorates CNS involvement in murine model of GM1-gangliosidosis
|
Tsunogai, Toshiki
|
|
|
25 |
C |
p. 448-460
|
artikel
|
| 23 |
Hexon modification of human adenovirus type 5 vectors enables efficient transduction of human multipotent mesenchymal stromal cells
|
Nilson, Robin
|
|
|
25 |
C |
p. 96-110
|
artikel
|
| 24 |
Hypo-glycosylated follistatin-like 1 for new cardiomyocyte formation
|
Kerkelä, Risto
|
|
|
25 |
C |
p. 331-332
|
artikel
|
| 25 |
Hypoxia-directed tumor targeting of CRISPR-Cas9 and HSV-TK suicide gene therapy using lipid nanoparticles
|
Davis, Alicia
|
|
|
25 |
C |
p. 158-169
|
artikel
|
| 26 |
Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation
|
Poletto, Edina
|
|
|
25 |
C |
p. 392-409
|
artikel
|
| 27 |
In vivo CRISPR-Cas9 inhibition of hepatic LDH as treatment of primary hyperoxaluria
|
Martinez-Turrillas, Rebeca
|
|
|
25 |
C |
p. 137-146
|
artikel
|
| 28 |
In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9
|
Hillen, Anne E.J.
|
|
|
25 |
C |
p. 17-25
|
artikel
|
| 29 |
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
|
Liang, Qiushi
|
|
|
25 |
C |
p. 520-532
|
artikel
|
| 30 |
Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler
|
Jin, Xiu
|
|
|
25 |
C |
p. 370-381
|
artikel
|
| 31 |
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice
|
Zieger, Marina
|
|
|
25 |
C |
p. 425-438
|
artikel
|
| 32 |
MSD-based assays facilitate a rapid and quantitative serostatus profiling for the presence of anti-AAV antibodies
|
Haar, Janina
|
|
|
25 |
C |
p. 360-369
|
artikel
|
| 33 |
Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy
|
Meng, Jinhong
|
|
|
25 |
C |
p. 491-507
|
artikel
|
| 34 |
Parallel Induction of CH505 B Cell Ontogeny-Guided Neutralizing Antibodies and tHIVconsvX Conserved Mosaic-Specific T Cells against HIV-1
|
Wee, Edmund G.
|
|
|
25 |
C |
p. 490
|
artikel
|
| 35 |
Pharmacological interventions enhance virus-free generation of TRAC-replaced CAR T cells
|
Kath, Jonas
|
|
|
25 |
C |
p. 311-330
|
artikel
|
| 36 |
Pre-clinical data supporting immunotherapy for HIV using CMV-HIV-specific CAR T cells with CMV vaccine
|
Guan, Min
|
|
|
25 |
C |
p. 344-359
|
artikel
|
| 37 |
Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants
|
Xi, Zhouhuan
|
|
|
25 |
C |
p. 476-489
|
artikel
|
| 38 |
Reversal of neuroinflammation in novel GS model mice by single i.c.v. administration of CHO-derived rhCTSA precursor protein
|
Horii, Yuto
|
|
|
25 |
C |
p. 297-310
|
artikel
|
| 39 |
Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors
|
Li, Chang
|
|
|
25 |
C |
p. 533
|
artikel
|
| 40 |
Self-amplifying mRNA SARS-CoV-2 vaccines raise cross-reactive immune response to variants and prevent infection in animal models
|
Palladino, Giuseppe
|
|
|
25 |
C |
p. 225-235
|
artikel
|
| 41 |
Tacrolimus-resistant SARS-CoV-2-specific T cell products to prevent and treat severe COVID-19 in immunosuppressed patients
|
Peter, Lena
|
|
|
25 |
C |
p. 52-73
|
artikel
|
| 42 |
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations
|
Mendell, Jerry R.
|
|
|
25 |
C |
p. 74-83
|
artikel
|
| 43 |
Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis
|
Sala, Davide
|
|
|
25 |
C |
p. 170-189
|
artikel
|
| 44 |
The ratio of nicotinic acid to nicotinamide as a microbial biomarker for assessing cell therapy product sterility
|
Huang, Jiayi
|
|
|
25 |
C |
p. 410-424
|
artikel
|
| 45 |
Transcriptomic analysis reveals optimal cytokine combinations for SARS-CoV-2-specific T cell therapy products
|
Durkee-Shock, Jessica
|
|
|
25 |
C |
p. 439-447
|
artikel
|
| 46 |
Treatment of experimental autoimmune encephalomyelitis using AAV gene therapy by blocking T cell costimulatory pathways
|
Zhong, Chen
|
|
|
25 |
C |
p. 461-475
|
artikel
|
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