Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

(2023) Sharla M. Birch et al.   Science Translational Medicine

Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence

(2022) Arianna Manini et al.   Frontiers in Neurology

Regulation of the myoblast fusion reaction for muscle development, regeneration, and adaptations

(2022) Douglas P. Millay   EXPERIMENTAL CELL RESEARCH

Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?

(2022) Takashi Kei Kishimoto et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Depletion of skeletal muscle satellite cells attenuates pathology in muscular dystrophy

(2022) Justin G. Boyer et al.   Nature Communications

The regenerating skeletal muscle niche drives satellite cell return to quiescence

(2022) Alicia A. Cutler et al.   iScience

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

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

(2021) Mohammadsharif Tabebordbar et al.   CELL

Extracellular vesicles as a next-generation drug delivery platform

(2021) Inge Katrin Herrmann et al.   Nature Nanotechnology

Changes in Myonuclear Number During Postnatal Growth –Implications for AAV Gene Therapy for Muscular Dystrophy

(2021) Jennifer Morgan et al.   Journal of Neuromuscular Diseases

One in five patients with Duchenne muscular dystrophy dies from other causes than cardiac or respiratory failure

(2021) Lisa Wahlgren et al.   EUROPEAN JOURNAL OF EPIDEMIOLOGY

Sarcomeric deficits underlie MYBPC1-associated myopathy with myogenic tremor

(2021) Janelle Geist Hauserman et al.   JCI Insight

Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV

(2020) Leon Morales et al.   MOLECULAR THERAPY

Myocyte-derived Myomaker expression is required for regenerative fusion but exacerbates membrane instability in dystrophic myofibers

(2020) Michael J. Petrany et al.   JCI Insight

Quantification of SARS-CoV-2 neutralizing antibody by a pseudotyped virus-based assay

(2020) Jianhui Nie et al.   Nature Protocols

Pseudotyping Lentiviral Vectors: When the Clothes Make the Virus

(2020) Alexis Duvergé et al.   Viruses-Basel

Human myotube formation is determined by MyoD–Myomixer/Myomaker axis

(2020) Haifeng Zhang et al.   Science Advances

Development of Novel Micro-dystrophins with Enhanced Functionality

(2019) Julian N. Ramos et al.   MOLECULAR THERAPY

How cells fuse

(2019) Nicolas G. Brukman et al.   JOURNAL OF CELL BIOLOGY

Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

(2019) Michela Milani et al.   Science Translational Medicine

AAV-based gene therapies for the muscular dystrophies

(2019) Julie M Crudele et al.   HUMAN MOLECULAR GENETICS

Fusogenic Reoviruses and Their Fusion-Associated Small Transmembrane (FAST) Proteins

(2019) Roy Duncan   Annual Review of Virology

The fusogenic synapse at a glance

(2019) Ji Hoon Kim et al.   JOURNAL OF CELL SCIENCE

Fusogens

(2018) Nadav Segev et al.   CURRENT BIOLOGY

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Shedding light on the cell biology of extracellular vesicles

(2018) Guillaume van Niel et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Myomaker and Myomerger Work Independently to Control Distinct Steps of Membrane Remodeling during Myoblast Fusion

(2018) Evgenia Leikina et al.   DEVELOPMENTAL CELL

In vivo myomaker-mediated heterologous fusion and nuclear reprogramming

(2017) Yasuyuki Mitani et al.   FASEB JOURNAL

ArabidopsisHAP2/GCS1 is a gamete fusion protein homologous to somatic and viral fusogens

(2017) Clari Valansi et al.   JOURNAL OF CELL BIOLOGY

Control of muscle formation by the fusogenic micropeptide myomixer

(2017) Pengpeng Bi et al.   SCIENCE

MyD88 promotes myoblast fusion in a cell-autonomous manner

(2017) Sajedah M. Hindi et al.   Nature Communications

The microprotein Minion controls cell fusion and muscle formation

(2017) Qiao Zhang et al.   Nature Communications

Myomerger induces fusion of non-fusogenic cells and is required for skeletal muscle development

(2017) Malgorzata E. Quinn et al.   Nature Communications

Requirement of myomaker-mediated stem cell fusion for skeletal muscle hypertrophy

(2017) Qingnian Goh et al.   eLife

Risks Associated With Lentiviral Vector Exposures and Prevention Strategies

(2016) Ryan Schlimgen et al.   JOURNAL OF OCCUPATIONAL AND ENVIRONMENTAL MEDICINE

Targeted, homology-driven gene insertion in stem cells by ZFN-loaded ‘all-in-one’ lentiviral vectors

(2016) Yujia Cai et al.   eLife

Established cell surface markers efficiently isolate highly overlapping populations of skeletal muscle satellite cells by fluorescence-activated cell sorting

(2016) Claire C. Maesner et al.   Skeletal Muscle

Pervasive satellite cell contribution to uninjured adult muscle fibers

(2015) Bradley Pawlikowski et al.   Skeletal Muscle

Myomaker is essential for muscle regeneration

(2014) Douglas P. Millay et al.   GENES & DEVELOPMENT

Myomaker is a membrane activator of myoblast fusion and muscle formation

(2013) Douglas P. Millay et al.   NATURE

A public genome-scale lentiviral expression library of human ORFs

(2011) Xiaoping Yang et al.   NATURE METHODS

Conserved Eukaryotic Fusogens Can Fuse Viral Envelopes to Cells

(2011) O. Avinoam et al.   SCIENCE

Lentiviral Vectors in Gene Therapy: Their Current Status and Future Potential

(2010) David Escors et al.   ARCHIVUM IMMUNOLOGIAE ET THERAPIAE EXPERIMENTALIS

Generation of VSV pseudotypes using recombinant ΔG-VSV for studies on virus entry, identification of entry inhibitors, and immune responses to vaccines

(2010) Michael A. Whitt   JOURNAL OF VIROLOGICAL METHODS

Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression

(2009) En Kimura et al.   MOLECULAR THERAPY

Mechanisms of membrane fusion: disparate players and common principles

(2008) Sascha Martens et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY