bioRxiv (Cold Spring Harbor Laboratory), Journal Year: 2024, Volume and Issue: unknown
Published: Nov. 11, 2024
Genetic vascular disorders are prevalent diseases that have diverse etiologies and few treatment options. Pathogenic missense mutations in the alpha actin isotype 2 gene (
Language: Английский
Journal of Controlled Release, Journal Year: 2024, Volume and Issue: 370, P. 763 - 772
Published: May 17, 2024
Language: Английский
Citations
17
Molecular Therapy — Nucleic Acids, Journal Year: 2025, Volume and Issue: 36(1), P. 102457 - 102457
Published: Jan. 18, 2025
Language: Английский
Citations
2
Journal of Controlled Release, Journal Year: 2024, Volume and Issue: 375, P. 366 - 388
Published: Sept. 18, 2024
Recent advancements in RNA therapeutics highlight the critical need for precision gene delivery systems that target specific organs and cells. Lipid nanoparticles (LNPs) have emerged as key vectors delivering mRNA siRNA, offering protection against enzymatic degradation, enabling targeted cellular uptake, facilitating cargo release into cytosol. This review discusses development optimization of organ- cell-specific LNPs, focusing on their design, mechanisms action, therapeutic applications. We explore innovations such DNA/RNA barcoding, which facilitates high-throughput screening precise adjustments formulations. address major challenges, including improving endosomal escape, minimizing off-target effects, enhancing efficiencies. Notable clinical trials recent FDA approvals illustrate practical applications future potential LNP-based therapies. Our findings suggest while considerable progress has been made, continued research is essential to resolve existing limitations bridge gap between pre-clinical evaluation safety efficacy therapeutics. highlights dynamic LNP research. It outlines a roadmap RNA-based medicine.
Language: Английский
Citations
14
Proceedings of the National Academy of Sciences, Journal Year: 2024, Volume and Issue: 121(11)
Published: March 4, 2024
Due to its small size and lifelong optical transparency, the fish Danionella cerebrum is an emerging model organism in biomedical research. How can this vertebrate under 12 mm length produce sounds over 140 dB? We found that it possesses ...Motion basis of nearly all animal behavior. Evolution has led some extraordinary specializations propulsion mechanisms among invertebrates, including mandibles dracula ant claw pistol shrimp. In contrast, ...
Language: Английский
Citations
6
Nano Today, Journal Year: 2025, Volume and Issue: 61, P. 102654 - 102654
Published: Jan. 31, 2025
Language: Английский
Citations
0
Nature Biotechnology, Journal Year: 2025, Volume and Issue: unknown
Published: March 20, 2025
Abstract Cell-type-specific regulatory elements such as enhancers can direct expression of recombinant adeno-associated viruses (AAVs) to specific cell types, but this approach is limited by the relatively small packaging capacity AAVs. In study, we used spatial genomics show that transcriptional crosstalk between individual AAV genomes provides a general method for cell-type-specific large cargo separating distally acting into second genome. We identified and profiled in carrying 11 different active mouse brain. developed methods identify localize their concatemeric forms cultured cells tissue, demonstrate here dependent upon concatemer formation. Finally, leveraged drive 3.2-kb Cas9 manner with systemically administered engineered AAVs, AAV-delivered, minimally invasive, gene editing wild-type mice recapitulates known disease phenotypes.
Language: Английский
Citations
0
Methods in molecular biology, Journal Year: 2025, Volume and Issue: unknown, P. 221 - 238
Published: Jan. 1, 2025
Language: Английский
Citations
0
Gene Therapy, Journal Year: 2025, Volume and Issue: unknown
Published: April 22, 2025
Language: Английский
Citations
0
Pharmaceutics, Journal Year: 2025, Volume and Issue: 17(5), P. 555 - 555
Published: April 24, 2025
Background: Advancements in pharmacogenomics, artificial intelligence (AI), and CRISPR gene-editing technology are revolutionizing precision medicine by enabling highly individualized therapeutic strategies. Artificial intelligence-driven computational techniques improve biomarker discovery drug optimization while pharmacogenomics helps to identify genetic polymorphisms affecting metabolism, efficacy, toxicity. Genetically editing based on presents a precise method for changing gene expression repairing damaging mutations. This review explores the convergence of these three fields enhance improved medicine. Method: A methodical study current literature was performed effects response variability, intelligence, predictive modeling applications. Results: Driven allows clinicians classify patients select appropriate medications depending their DNA profiles. reduces side effect risk increases efficacy. Precision modifications made feasible therapy outcomes oncology, metabolic illnesses, neurological diseases, other fields. The integration streamlines genome-editing applications, lowers off-target effects, specificity. Notwithstanding advances, issues including biases, moral dilemmas, legal constraints still arise. Conclusions: synergy alters letting customized interventions. Clinically translating, however, hinges resolving data privacy concerns, assuring equitable access, strengthening systems. Future research should focus refining technologies, enhancing AI-driven developing guidelines applying tools going forward.
Language: Английский
Citations
0
Human Gene Therapy, Journal Year: 2025, Volume and Issue: unknown
Published: April 28, 2025
The advent of genome editing has kindled the hope to cure previously uncurable, life-threatening genetic diseases. However, whether this promise can be ultimately fulfilled depends on how efficiently gene agents delivered therapeutically relevant cells. Over time, viruses have evolved into sophisticated, versatile, and biocompatible nanomachines that engineered shuttle payloads specific cell types. Despite advances in safety selectivity, long-term expression sustained by viral vectors remains a cause for concern. Cell-derived vesicles (CDVs) are gaining traction as elegant alternatives. CDVs encompass extracellular (EVs), diverse set intrinsically low-immunogenic membranous nanoparticles, virus-like particles (VLPs), bioparticles with scaffold envelope structures, but devoid material. Both EVs VLPs deliver ribonucleoprotein cargo target cytoplasm, ensuring machinery is only transiently active thereby increasing its safety. In review, we explore natural diversity their potential delivery clustered regularly interspaced short palindromic repeats (CRISPR) machinery. We illustrate different strategies optimization CDV loading retargeting, highlighting versatility tunability these vehicles. Nonetheless, lack robust standardized protocols production, purification, quality assessment still hinders widespread adoption further CRISPR-based therapies advanced "living drugs." believe collective, multifaceted effort urgently needed address critical issues unlock full genome-editing technologies yield safe, easy-to-manufacture, pharmacologically well-defined therapies. Finally, discuss current clinical landscape lung-directed cystic fibrosis could drive significant breakthroughs vivo disease.
Language: Английский
Citations
0