Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

Allaire, Norm; Griesenbach, Uta; Kerem, Batsheva; Lueck, John D.; Stanleigh, Noemie; Oren, Yifat S.

doi:10.1016/j.jcf.2022.12.016

Cited by 10 publications

(2 citation statements)

References 37 publications

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“…Among gene therapy technologies, antisense oligonucleotides (ASOs) represent another area of RNA application. These short nucleotide sequences, complementary to specific mRNA fragments, can effectively inhibit translational processes or initiate protein synthesis, leading to the blockade of a particular gene expression [ 142 ]. An up-and-coming field also uses RNA itself, namely mRNA, as a potential therapeutic agent.…”

Section: Nucleic-acid-based Drug Delivery Systemsmentioning

confidence: 99%

Biomaterials in Drug Delivery: Advancements in Cancer and Diverse Therapies—Review

Drabczyk,

Kudłacik-Kramarczyk,

Jamroży

et al. 2024

IJMS

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Nano-sized biomaterials are innovative drug carriers with nanometric dimensions. Designed with biocompatibility in mind, they enable precise drug delivery while minimizing side effects. Controlled release of therapeutic substances enhances efficacy, opening new possibilities for treating neurological and oncological diseases. Integrated diagnostic-therapeutic nanosystems allow real-time monitoring of treatment effectiveness, which is crucial for therapy personalization. Utilizing biomaterials as nano-sized carriers in conjunction with drugs represents a promising direction that could revolutionize the field of pharmaceutical therapy. Such carriers represent groundbreaking drug delivery systems on a nanometric scale, designed with biocompatibility in mind, enabling precise drug delivery while minimizing side effects. Using biomaterials in synergy with drugs demonstrates significant potential for a revolutionary impact on pharmaceutical therapy. Conclusions drawn from the review indicate that nano-sized biomaterials constitute an innovative tool that can significantly improve therapy effectiveness and safety, especially in treating neurological and oncological diseases. These findings should guide researchers towards further studies to refine nano-sized biomaterials, assess their effectiveness under various pathological conditions, and explore diagnostic-therapeutic applications. Ultimately, these results underscore the promising nature of nano-sized biomaterials as advanced drug carriers, ushering in a new era in nanomedical therapy.

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Section: Nucleic-acid-based Drug Delivery Systemsmentioning

confidence: 99%

Biomaterials in Drug Delivery: Advancements in Cancer and Diverse Therapies—Review

Drabczyk,

Kudłacik-Kramarczyk,

Jamroży

et al. 2024

IJMS

View full text Add to dashboard Cite

show abstract

“…therapeutics into the cell. (24)(25)(26)(27)(28)(29) To improve intracellular uptake, phage display can be useful for selecting targeting ligands with affinity for cells and specific tissues. We previously used T7 phage combinatorial libraries against a CF-like mucus model and screened for peptides that showed enhanced transport through CF patient-derived sputum.…”

Section: Selected Peptide Functionalized Lnps Enhance Mrna Expression...mentioning

confidence: 99%

Discovery of peptides for targeted delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia

Soto,

Lewis,

Leal

et al. 2023

Preprint

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For cystic fibrosis (CF) patients, a lung targeted gene therapy would significantly alleviate pulmonary complications associated with morbidity and mortality. However, mucus in the airways and cell entry pose huge delivery barriers for local gene therapy. Here, we used phage display technology to select for and identify mucus- and cell-penetrating peptides against primary human bronchial epithelial cells (pHBECs) from CF patients cultured at air-liquid interface (ALI). At ALI, pHBECs produce mucus and reflect CF disease pathology, making it a clinically relevant model. Using this model, we discovered a lead candidate peptide, and incorporated it into lipid nanoparticles (LNPs) to deliver mRNA to pHBECs and mouse lungsin vivo. Compared to LNPs without our peptide, peptide-LNPs demonstrated 7.8-fold and 4.8-fold higher mRNA expressionin vitroandin vivo, respectively. Since gene delivery to pHBECs is a significant challenge, we are encouraged by these results and anticipate that our peptide could be used to successfully deliver CF gene therapies in future work.

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Cystic Fibrosis—A success story in pediatrics

Dittrich

2023

Monatsschr Kinderheilkd

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Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

Cited by 10 publications

References 37 publications

Biomaterials in Drug Delivery: Advancements in Cancer and Diverse Therapies—Review

Biomaterials in Drug Delivery: Advancements in Cancer and Diverse Therapies—Review

Discovery of peptides for targeted delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia

Cystic Fibrosis—A success story in pediatrics

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