Effects of CRISPR-Cas9-mediated <em>FOXP3</em> knockout on CAR T cell potency
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Molecular Therapy Methods & Clinical Development
- Publisher:
- Elsevier
- ISSN:
- 2329-0501
- Category:
- MEDICINE, RESEARCH & EXPERIMENTAL
- Impact factor:
- 4.6
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32 parsed articles
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A potency and toxicology study on tolerability of cryopreserved mesenchymal stem cell product with DMSO in septic mice and nude rats
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Retinal organoids mirror CRISPR-Cas9 gene editing efficiency observed <em>in vivo</em>
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SLC20A1 knockout to stably express a fusogenic gibbon ape leukemia virus envelope glycoprotein for lentiviral vector production
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<em>In vivo</em> delivery of a fluorogenic antisense oligonucleotide into human leukocytes in a humanized mouse model system
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Wild-type and engineered adeno-associated viral vectors produce comparable opsin expression and light-evoked responses in rat skeletal muscle
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Development of LC-MS methods for AAV capsid protein quantification and host cell protein profiling
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Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy
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Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia
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A rescue fanconi anemia humanized mouse model with endogenous FA mutation and high human hematopoietic stem cell chimerism
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Reduced rAAV interference enhances rcAAV detection sensitivity
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Bedside manufacturing of engineered stem cells using gene therapy foam
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Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: Insights into organ toxicities
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AAV-PHP.eB achieves superior neuronal transduction over AAV9 in pigtail macaques following intracerebroventricular administration
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Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study
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Efficient kidney gene transfer and proximal tubule transduction using self-complementary AAV.cc47 vectors
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The amazing AAV capsids: Into the structure-verse
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Simple, specific, rapid, and pharmacopoeia-compliant qPCR approach for the detection of mycoplasma in biopharmaceuticals
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Are viral vector-mediated therapies compatible with aberrant glycosylation?
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Targeted inhibition of <em>ELANE</em> expression using adenine base editing to treat severe congenital neutropenia
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High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa
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HBVZ10, an AAV8 vector-based new HBV therapy candidate for cccDNA elimination
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Heavy-chain immunoglobulin locus editing in rhesus macaque B cells to confer antibody production
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Directed evolution of liver-detargeted AAV vectors for systemic gene delivery to skeletal muscle and heart
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Chronologically distributed transfection improves AAV2 and AAV2/8 capsid filling and reveals assembly schedule divergence
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From cold chain to ambient: Benefits, risks, and evidence across cell therapy logistics
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Therapeutic mRNA delivery of CRISPR-Cas9 to the trabecular meshwork reverses ocular hypertension in myocilin glaucoma
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Variation of VP2 stoichiometry and deamidation of VP1 during production and their impacts on the transduction efficiency of AAV vectors
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Establishment of a novel human amniotic epithelial-derived cell line, HAT, for high-yield AAV vector production
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Design and systematic evaluation of novel chemical modification patterns enable the identification of a potent siRNA targeting <em>PCSK9</em>
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