Michael J. Mitchell

Next-generation experimental techniques for mRNA lipid nanoparticle evaluation and protein interaction analysis

The promise of RNA therapeutics is tanta-lizing to drug delivery scientists, as RNA-en-coded genes generally need only reach the cytoplasm to perform their therapeutic functions, circumventing the need for nuclear localization to achieve transcription. 1 However, targeted intracellular delivery of mRNA is difficult, re...

Functional Improvement of dystrophic muscle by utrophin gene editing

Nanomedicine beyond carriers — devices, cells & living therapeutics

This perspective summarizes the current landscape of biomedical nanotechnologies and outlines how their continued evolution positions nanomedicine as an enabling science driving the next generation of therapeutics.

Structural evolution of ionizable lipids for nucleic acid delivery

This Review provides insights into the challenges and gaps for translation, manufacturing and expansion of lipid nanoparticle-mediated nucleic acid therapeutics for precision RNA medicine while highlighting next-generation principles and translational perspectives that may guide precision RNA therapeutics in regenerati...

Resolving Heterogeneity of Targeted Lipid Nanoparticles Through Solution‐Based Biophysical Analyses

This work harnesses a separation‐coupled biophysical platform to resolve previously inaccessible tLNP subpopulations and provides insights to inform rational engineering of next‐generation targeted RNA therapeutics.

Transient T cell therapies.

The pharmacological imperative for transient CAR expression is delineated, including lipid nanoparticles in combination with mRNA, which avoids risks of insertional mutagenesis and enables titratable, short-lived CAR expression, thereby enhancing safety management and suitability for applications beyond oncology.

Targeted lipid nanoparticles unlock in vivo human haematopoietic stem cell gene editing.