References

Skeletal muscle atrophy and dysfunction in obesity and type-2 diabetes mellitus: Myocellular mechanisms involved

10.1007/s11154-025-09954-9 · 2025 · External reference

Integrative Approaches to Myopathies and Muscular Dystrophies: Molecular Mechanisms, Diagnostics, and Future Therapies

10.3390/ijms26167972 · 2025 · External reference

Rosa26-LSL-dCas9-VPR: a versatile mouse model for tissue specific and simultaneous activation of multiple genes for drug discovery

10.1038/s41598-022-23127-7 · 2022 · External reference

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

10.1016/j.cell.2020.03.023 · 2020 · External reference

Modeling Pulmonary Disease Pathways Using Recombinant Adeno-Associated Virus 6.2

10.1165/rcmb.2014-0338ma · 2015 · External reference

Induction of Sustained Hypercholesterolemia by Single Adeno-Associated Virus–Mediated Gene Transfer of Mutant hPCSK9

10.1161/atvbaha.114.303617 · 2015 · External reference

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

10.1089/hum.2009.086 · 2009 · External reference

Rapid and highly efficient inducible cardiac gene knockout in adult mice using AAV-mediated expression of Cre recombinase

10.1093/cvr/cvu174 · 2014 · External reference

Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems

10.1016/j.neuron.2022.05.003 · 2022 · External reference

Adipose tissue insulin receptor knockdown via a new primate-derived hybrid recombinant AAV serotype

10.1038/mtm.2013.8 · 2014 · External reference

Mapping administration route-dependent transduction profiles of commonly used AAV variants in mice by barcode amplicon sequencing

10.1016/j.omtm.2025.101468 · 2025 · External reference

Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

10.1038/s41467-020-19230-w · 2020 · External reference

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

10.1016/j.cell.2021.08.028 · 2021 · External reference

Comparative in vivo characterization of newly discovered myotropic adeno-associated vectors

10.1186/s13395-024-00341-7 · 2024 · External reference

An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species

10.1038/s41467-024-52002-4 · 2024 · External reference

Muscle-Specific Promoters for Gene Therapy

10.32607/actanaturae.11063 · 2021 · External reference

Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library

10.1016/j.ymthe.2025.04.027 · 2025 · External reference

Assessment of Tropism and Effectiveness of New Primate-Derived Hybrid Recombinant AAV Serotypes in the Mouse and Primate Retina

10.1371/journal.pone.0060361 · 2013 · External reference

Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy

10.1016/j.omtm.2022.09.014 · 2022 · External reference

Targeting Visceral Fat by Intraperitoneal Delivery of Novel AAV Serotype Vector Restricting Off-Target Transduction in Liver

10.1016/j.omtm.2017.06.002 · 2017 · External reference

Gene Delivery to Adipose Tissue Using Transcriptionally Targeted rAAV8 Vectors

10.1371/journal.pone.0116288 · 2014 · External reference

Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans

10.1016/0092-8674(93)90530-4 · 1993 · External reference

The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14

10.1016/0092-8674(93)90529-y · 1993 · External reference

Argonaute2 Is the Catalytic Engine of Mammalian RNAi

10.1126/science.1102513 · 2004 · External reference

A microRNA in a Multiple-Turnover RNAi Enzyme Complex

10.1126/science.1073827 · 2002 · External reference

Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells

10.1016/j.omtm.2024.101280 · 2024 · External reference

miR-122, a Mammalian Liver-Specific microRNA, is Processed from hcr mRNA and MayDownregulate the High Affinity Cationic Amino Acid Transporter CAT-1

10.4161/rna.1.2.1066 · 2004 · External reference

Development of an Optimized Promoter System for Exosomal and Naked AAV Vector-Based Suicide Gene Therapy in Hepatocellular Carcinoma

10.1021/acsomega.4c03949 · 2024 · External reference

Construction and analysis of compact muscle-specific promoters for AAV vectors

10.1038/gt.2008.104 · 2008 · External reference

Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences

10.1038/6981 · 1999 · External reference

Next-generation muscle-directed gene therapy by in silico vector design

10.1038/s41467-018-08283-7 · 2019 · External reference

miRNATissueAtlas 2025: an update to the uniformly processed and annotated human and mouse non-coding RNA tissue atlas

10.1093/nar/gkae1036 · 2025 · External reference

A Family of microRNAs Encoded by Myosin Genes Governs Myosin Expression and Muscle Performance

10.1016/j.devcel.2009.10.013 · 2009 · External reference

Targeting adipose tissue via systemic gene therapy

10.1038/gt.2014.38 · 2014 · External reference

Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver

10.1038/gt.2010.157 · 2011 · External reference

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

10.1038/mt.2010.279 · 2011 · External reference

Application of Mutated miR-206 Target Sites Enables Skeletal Muscle-specific Silencing of Transgene Expression of Cardiotropic AAV9 Vectors

10.1038/mt.2012.276 · 2013 · External reference

Regulation of expression of the mouse adiponectin gene by the C/EBP family via a novel enhancer region

10.1016/j.gene.2008.07.039 · 2008 · External reference

In Vivo Adeno-Associated Viral Vector–Mediated Genetic Engineering of White and Brown Adipose Tissue in Adult Mice

10.2337/db13-0311 · 2013 · External reference

FGF6 and FGF9 regulate UCP1 expression independent of brown adipogenesis

10.1038/s41467-020-15055-9 · 2020 · External reference

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

10.1038/gt.2010.141 · 2011 · External reference

Restriction of Calpain3 Expression to the Skeletal Muscle Prevents Cardiac Toxicity and Corrects Pathology in a Murine Model of Limb-Girdle Muscular Dystrophy

10.1161/circulationaha.113.001340 · 2013 · External reference

Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss

10.1016/j.ymthe.2024.06.036 · 2024 · External reference

Treatment of multifocal breast cancer by systemic delivery of dual-targeted adeno-associated viral vectors

10.1038/gt.2015.52 · 2015 · External reference

AAV- based vector improvements unrelated to capsid protein modification

10.3389/fmed.2023.1106085 · 2023 · External reference

Have a little heart (or not): highly minimized skeletal muscle regulatory cassettes with low or no activity in heart

10.1089/hum.2024.041 · 2024 · External reference

siRNAs can function as miRNAs

10.1101/gad.1064703 · 2003 · External reference

A toolbox of astrocyte-specific, serotype-independent adeno-associated viral vectors using microRNA targeting sequences

10.1038/s41467-023-42746-w · 2023 · External reference

Standardized, Scalable, and Timely Flexible Adeno-Associated Virus Vector Production Using Frozen High-Density HEK-293 Cell Stocks and CELLdiscs

10.1089/hgtb.2018.228 · 2019 · External reference

AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements

10.1016/j.omtm.2025.101499 · 2025 · External reference