| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
A high-fidelity RNA-targeting Cas13 restores paternal Ube3a expression and improves motor functions in Angelman syndrome mice
|
Li, Jinhui
|
|
|
31 |
7 |
p. 2286-2295
|
artikel
|
| 2 |
Always a bridesmaid, never a bride: Committing to flu neuraminidase as a vaccine target
|
Schneider, Cosette
|
|
|
31 |
7 |
p. 1865-1867
|
artikel
|
| 3 |
An optimized SpCas9 high-fidelity variant for direct protein delivery
|
Pedrazzoli, Eleonora
|
|
|
31 |
7 |
p. 2257-2265
|
artikel
|
| 4 |
ASGCT 2023—Gene therapy is becoming medicine
|
Herzog, Roland W.
|
|
|
31 |
7 |
p. 1859
|
artikel
|
| 5 |
A trans-amplifying RNA simplified to essential elements is highly replicative and robustly immunogenic in mice
|
Perkovic, Mario
|
|
|
31 |
7 |
p. 2297
|
artikel
|
| 6 |
Augmenting TCR signal strength and ICOS costimulation results in metabolically fit and therapeutically potent human CAR Th17 cells
|
Wyatt, Megan M.
|
|
|
31 |
7 |
p. 2120-2131
|
artikel
|
| 7 |
Beyond the unfolded protein response: Disclosing the role of XBP1s in Alzheimer’s disease
|
Marcello, Elena
|
|
|
31 |
7 |
p. 1868-1869
|
artikel
|
| 8 |
Biomaterial-enabled therapeutic modulation of cGAS-STING signaling for enhancing antitumor immunity
|
Liu, Yingqi
|
|
|
31 |
7 |
p. 1938-1959
|
artikel
|
| 9 |
Chimeric TIM-4 receptor-modified T cells targeting phosphatidylserine mediates both cytotoxic anti-tumor responses and phagocytic uptake of tumor-associated antigen for T cell cross-presentation
|
Cieniewicz, Brandon
|
|
|
31 |
7 |
p. 2132-2153
|
artikel
|
| 10 |
Clinical perspective: Antibody-drug conjugates for the treatment of HER2-positive breast cancer
|
Najminejad, Zohreh
|
|
|
31 |
7 |
p. 1874-1903
|
artikel
|
| 11 |
CRISPR-Cas-mediated transcriptional modulation: The therapeutic promises of CRISPRa and CRISPRi
|
Bendixen, Louise
|
|
|
31 |
7 |
p. 1920-1937
|
artikel
|
| 12 |
CTLA-4 blockade induces tumor pyroptosis via CD8+ T cells in head and neck squamous cell carcinoma
|
Wang, Shuo
|
|
|
31 |
7 |
p. 2154-2168
|
artikel
|
| 13 |
Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa
|
Occelli, Laurence M.
|
|
|
31 |
7 |
p. 2028-2041
|
artikel
|
| 14 |
Dual targeting of CD19 and CD22 against B-ALL using a novel high-sensitivity aCD22 CAR
|
Kokalaki, Evangelia
|
|
|
31 |
7 |
p. 2089-2104
|
artikel
|
| 15 |
Extracellular communication between brain cells through functional transfer of Cre mRNA mediated by extracellular vesicles
|
Rufino-Ramos, David
|
|
|
31 |
7 |
p. 2220-2239
|
artikel
|
| 16 |
Gene therapy advances shine the spotlight on epidermolysis bullosa, bringing hope to patients
|
Koller, Ulrich
|
|
|
31 |
7 |
p. 1860-1861
|
artikel
|
| 17 |
In this issue
|
|
|
|
31 |
7 |
p. 1864
|
artikel
|
| 18 |
Learning to lock down genetic diseases without throwing away the key
|
Tai, Phillip W.L.
|
|
|
31 |
7 |
p. 1862-1863
|
artikel
|
| 19 |
Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson’s disease
|
Rocco, Matthew T.
|
|
|
31 |
7 |
p. 2296
|
artikel
|
| 20 |
Neuraminidase delivered as an APC-targeted DNA vaccine induces protective antibodies against influenza
|
Werninghaus, Ina Charlotta
|
|
|
31 |
7 |
p. 2188-2205
|
artikel
|
| 21 |
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma
|
Chitranshi, Nitin
|
|
|
31 |
7 |
p. 2056-2076
|
artikel
|
| 22 |
Ocular stress enhances contralateral transfer of lenadogene nolparvovec gene therapy through astrocyte networks
|
McGrady, Nolan R.
|
|
|
31 |
7 |
p. 2005-2013
|
artikel
|
| 23 |
Optimized CAR-T therapy based on spatiotemporal changes and chemotactic mechanisms of MDSCs induced by hypofractionated radiotherapy
|
Zhang, Benxia
|
|
|
31 |
7 |
p. 2105-2119
|
artikel
|
| 24 |
Phase I study of liver depot gene therapy in late-onset Pompe disease
|
Smith, Edward C.
|
|
|
31 |
7 |
p. 1994-2004
|
artikel
|
| 25 |
Polyphyllin D punctures hypertrophic lysosomes to reverse drug resistance of hepatocellular carcinoma by targeting acid sphingomyelinase
|
Wang, Yang
|
|
|
31 |
7 |
p. 2169-2187
|
artikel
|
| 26 |
Precise excision of HTLV-1 provirus with a designer-recombinase
|
Rojo-Romanos, Teresa
|
|
|
31 |
7 |
p. 2266-2285
|
artikel
|
| 27 |
Preclinical evaluation of ADVM-062, a novel intravitreal gene therapy vector for the treatment of blue cone monochromacy
|
Hanna, Kelly
|
|
|
31 |
7 |
p. 2014-2027
|
artikel
|
| 28 |
RAS degraders: The new frontier for RAS-driven cancers
|
Escher, Taylor E.
|
|
|
31 |
7 |
p. 1904-1919
|
artikel
|
| 29 |
Re-administration of AAV vectors by masking with host albumin: A Goldilocks hypothesis
|
Wright, J. Fraser
|
|
|
31 |
7 |
p. 1870-1873
|
artikel
|
| 30 |
Safety and dose escalation of the targeted oncolytic adenovirus OBP-301 for refractory advanced liver cancer: Phase I clinical trial
|
Heo, Jeong
|
|
|
31 |
7 |
p. 2077-2088
|
artikel
|
| 31 |
β-secretase 1 overexpression by AAV-mediated gene delivery prevents retina degeneration in a mouse model of age-related macular degeneration
|
Qi, Xiaoping
|
|
|
31 |
7 |
p. 2042-2055
|
artikel
|
| 32 |
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy
|
Logan, Grant J.
|
|
|
31 |
7 |
p. 1979-1993
|
artikel
|
| 33 |
Targeted therapy for rare lung cancers: Status, challenges, and prospects
|
Wang, Chunsen
|
|
|
31 |
7 |
p. 1960-1978
|
artikel
|
| 34 |
The unfolded protein response transcription factor XBP1s ameliorates Alzheimer’s disease by improving synaptic function and proteostasis
|
Duran-Aniotz, Claudia
|
|
|
31 |
7 |
p. 2240-2256
|
artikel
|
| 35 |
Tracking human neurologic disease status in mouse brain/plasma using reporter-tagged, EV-associated biomarkers
|
Maalouf, Katia E.
|
|
|
31 |
7 |
p. 2206-2219
|
artikel
|
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