| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency
|
Isgrig, Kevin
|
|
|
26 |
C |
p. 371-383
|
artikel
|
| 2 |
AAV-mediated rescue of Eps8 expression in vivo restores hair-cell function in a mouse model of recessive deafness
|
Jeng, Jing-Yi
|
|
|
26 |
C |
p. 355-370
|
artikel
|
| 3 |
AAV-monoclonal antibody expression protects mice from Ebola virus without impeding the endogenous antibody response to heterologous challenge
|
van Lieshout, Laura P.
|
|
|
26 |
C |
p. 505-518
|
artikel
|
| 4 |
Amelioration of experimental tendinopathy by lentiviral CD44 gene therapy targeting senescence-associated secretory phenotypes
|
Chen, Shih-Yao
|
|
|
26 |
C |
p. 157-168
|
artikel
|
| 5 |
Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action
|
Liu, Su
|
|
|
26 |
C |
p. 61-71
|
artikel
|
| 6 |
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system
|
Meng, Yuan
|
|
|
26 |
C |
p. 1-3
|
artikel
|
| 7 |
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy
|
Farrar, Michelle A.
|
|
|
26 |
C |
p. 96-97
|
artikel
|
| 8 |
Decrease in Angiotensin-Converting Enzyme activity but not concentration in plasma/lungs in COVID-19 patients offers clues for diagnosis/treatment
|
Daniell, Henry
|
|
|
26 |
C |
p. 266-278
|
artikel
|
| 9 |
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases
|
Oishi, Tomoya
|
|
|
26 |
C |
p. 253-265
|
artikel
|
| 10 |
Efficient production of inhibitor-free foamy virus glycoprotein-containing retroviral vectors by proteoglycan-deficient packaging cells
|
Munz, Clara Marie
|
|
|
26 |
C |
p. 394-412
|
artikel
|
| 11 |
Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in liver fibrosis
|
Bu, Fang-tian
|
|
|
26 |
C |
p. 191-206
|
artikel
|
| 12 |
Evaluation of parameters for efficient purification and long-term storage of herpes simplex virus-based vectors
|
Kuroda, Seiji
|
|
|
26 |
C |
p. 132-143
|
artikel
|
| 13 |
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice
|
Padula, Agnese
|
|
|
26 |
C |
p. 495-504
|
artikel
|
| 14 |
Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application
|
Kitano, Yuko
|
|
|
26 |
C |
p. 15-25
|
artikel
|
| 15 |
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2
|
Prabhakar, Shilpa
|
|
|
26 |
C |
p. 169-180
|
artikel
|
| 16 |
High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson’s disease
|
Murillo, Oihana
|
|
|
26 |
C |
p. 98-106
|
artikel
|
| 17 |
Immortalized human myoblast cell lines for the delivery of therapeutic proteins using encapsulated cell technology
|
Lathuiliere, Aurelien
|
|
|
26 |
C |
p. 441-458
|
artikel
|
| 18 |
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper
|
Yang, Tong-yuan
|
|
|
26 |
C |
p. 471-494
|
artikel
|
| 19 |
Impact of genetically modified organism requirements on gene therapy development in the EU, Japan, and the US
|
Tajima, Gentaro
|
|
|
26 |
C |
p. 74-83
|
artikel
|
| 20 |
In vivo generation of CAR T cells in the presence of human myeloid cells
|
Ho, Naphang
|
|
|
26 |
C |
p. 144-156
|
artikel
|
| 21 |
Liver gene therapy: The magic bullet for the sick lung
|
Brunetti-Pierri, Nicola
|
|
|
26 |
C |
p. 72-73
|
artikel
|
| 22 |
Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase
|
Tao, Rui
|
|
|
26 |
C |
p. 95
|
artikel
|
| 23 |
mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model
|
Cacicedo, Maximiliano L.
|
|
|
26 |
C |
p. 294-308
|
artikel
|
| 24 |
Mutant allele knockout with novel CRISPR nuclease promotes myelopoiesis in ELANE neutropenia
|
Sabo, Peter
|
|
|
26 |
C |
p. 119-131
|
artikel
|
| 25 |
Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS
|
Smith, Laura J.
|
|
|
26 |
C |
p. 224-238
|
artikel
|
| 26 |
PAM-altering SNP-based allele-specific CRISPR-Cas9 therapeutic strategies for Huntington’s disease
|
Shin, Jun Wan
|
|
|
26 |
C |
p. 547-561
|
artikel
|
| 27 |
PAM-flexible dual base editor-mediated random mutagenesis and self-activation strategies to improve CRISPRa potency
|
Lau, Cia-Hin
|
|
|
26 |
C |
p. 26-37
|
artikel
|
| 28 |
Plasmodium vivax latent liver infection is characterized by persistent hypnozoites, hypnozoite-derived schizonts, and time-dependent efficacy of primaquine
|
Flannery, Erika L.
|
|
|
26 |
C |
p. 427-440
|
artikel
|
| 29 |
Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I
|
Mesa-Núñez, Cristina
|
|
|
26 |
C |
p. 459-470
|
artikel
|
| 30 |
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene
|
Viker, Kimberly B.
|
|
|
26 |
C |
p. 532-546
|
artikel
|
| 31 |
Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa
|
Lenders, Malte
|
|
|
26 |
C |
p. 323-330
|
artikel
|
| 32 |
Quantification of transgene expression in GSH AAVS1 with a novel CRISPR/Cas9-based approach reveals high transcriptional variation
|
Inderbitzin, Anne
|
|
|
26 |
C |
p. 107-118
|
artikel
|
| 33 |
Selective B cell depletion upon intravenous infusion of replication-incompetent anti-CD19 CAR lentivirus
|
Rive, Craig M.
|
|
|
26 |
C |
p. 4-14
|
artikel
|
| 34 |
Sendai F/HN pseudotyped lentiviral vector transduces human ciliated and non-ciliated airway cells using α 2,3 sialylated receptors
|
Munday, Rosie J.
|
|
|
26 |
C |
p. 239-252
|
artikel
|
| 35 |
Single-dose AAV vector gene immunotherapy to treat food allergy
|
Gonzalez-Visiedo, Miguel
|
|
|
26 |
C |
p. 309-322
|
artikel
|
| 36 |
Structural basis for the neurotropic AAV9 and the engineered AAVPHP.eB recognition with cellular receptors
|
Xu, Guangxue
|
|
|
26 |
C |
p. 52-60
|
artikel
|
| 37 |
Structural basis of receptor usage by the engineered capsid AAV-PHP.eB
|
Jang, Seongmin
|
|
|
26 |
C |
p. 343-354
|
artikel
|
| 38 |
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
|
Wein, Nicolas
|
|
|
26 |
C |
p. 279-293
|
artikel
|
| 39 |
Targeted biallelic integration of an inducible Caspase 9 suicide gene in iPSCs for safer therapies
|
Wunderlich, Stephanie
|
|
|
26 |
C |
p. 84-94
|
artikel
|
| 40 |
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid
|
Goertsen, David
|
|
|
26 |
C |
p. 331-342
|
artikel
|
| 41 |
T cells isolated from G-CSF-treated multiple myeloma patients are suitable for the generation of BCMA-directed CAR-T cells
|
Battram, Anthony M.
|
|
|
26 |
C |
p. 207-223
|
artikel
|
| 42 |
Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency
|
Lam, Patricia
|
|
|
26 |
C |
p. 413-426
|
artikel
|
| 43 |
Thermostable H1 hemagglutinin stem with M2e epitopes provides broad cross-protection against group1 and 2 influenza A viruses
|
Subbbiah, Jeeva
|
|
|
26 |
C |
p. 38-51
|
artikel
|
| 44 |
Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery
|
Hunter, Jacqueline E.
|
|
|
26 |
C |
p. 384-393
|
artikel
|
| 45 |
Vaccination of cats with Sad23L-nCoV-S vaccine candidate against major variants of SARS-CoV-2
|
Zhang, Panli
|
|
|
26 |
C |
p. 181-190
|
artikel
|
| 46 |
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood
|
Zhang, Lening
|
|
|
26 |
C |
p. 519-531
|
artikel
|
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