FDA approves patisiran to treat hereditary transthyretin amyloidosis

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Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis

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RNA Therapeutics for Improving CAR T-cell Safety and Efficacy

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Transthyretin Stabilization: An Emerging Strategy for the Treatment of Alzheimer’s Disease?

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Diagnostic and Treatment Approaches Involving Transthyretin in Amyloidogenic Diseases

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Cyanuric chloride as the basis for compositionally diverse lipids

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How the initial discovery of modified RNA enabled evasion of innate immune responses and facilitated the development of RNA therapeutics

Sioud, M. (2023) · Scandinavian Journal of Immunology · doi:10.1111/sji.13282 · Cited by 7 · see citing works

IL-22: A key inflammatory mediator as a biomarker and potential therapeutic target for lung cancer

Xu, L., Cao, P., Wang, J., Zhang, P., Hu, S., Cheng, C., & Wang, H. (2024) · Heliyon · doi:10.1016/j.heliyon.2024.e35901 · Cited by 7 · see citing works

Small interfering RNA (siRNA) based therapies for cancer treatment: A special focus on cervical cancer

Bavishi, L., Butani, S., & Patel, S. S. (2025) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2025.178198 · Cited by 7 · see citing works

Localized RNA interference therapy to eliminate residual lung cancer after incomplete microwave ablation

Cao, F., Wan, C., Xie, L., Qi, H., Shen, L., Chen, S., Song, Z., & Fan, W. (2019) · Thoracic Cancer · doi:10.1111/1759-7714.13079 · Cited by 7 · see citing works

Exploring the Potentials of Antibody–siRNA Conjugates in Tumor Cell Gene Silencing without Cationic Assistance

Liu, Y., Quan, Y., Mao, Q., Xu, R., & Tai, W. (2025) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.5c00212 · Cited by 6 · see citing works

The Efficacy and Safety of Polyethylene Glycol Cholesterol- and Tocopherol Polyethylene Glycol 1000 Succinate-Modified Transforming Growth Factor β1 Small Interfering RNA Lipid Nanoparticles in the Treatment of Paclitaxel-Resistant Non-Small-Cell Lung Cancer

Zeng, Z., Zeng, X., Li, X., Feng, Y., Kan, Y., Liu, X., & Zeng, Y. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16010075 · Cited by 6 · see citing works

Innovative Genoceuticals in Human Gene Therapy Solutions: Challenges and Safe Clinical Trials of Orphan Gene Therapy Products

Sharma, R. K. (2023) · Current Gene Therapy · doi:10.2174/1566523223666230911120922 · Cited by 6 · see citing works

Which Nanobasics Should Be Taught in Medical Schools?

Sunshine, J. C., & Paller, A. S. (2019) · The AMA Journal of Ethic · doi:10.1001/amajethics.2019.337 · Cited by 6 · see citing works

A era dos medicamentos de ARN interferência: o panorama clínico dos fármacos para silenciamento génico

Godinho, B. M. D. C., & Khvorova, A. (2020) · Portuguese National Funding Agency for Science, Research and Technology (RCAAP Project by FCT) · doi:10.25758/set.2227 · Cited by 6 · see citing works

Nonviral vector system for cancer immunogene therapy

Nie, W., Chen, J., Wang, B., & Gao, X. (2022) · MedComm – Biomaterials and Applications · doi:10.1002/mba2.10 · Cited by 6 · see citing works

Real-Life Data of 2-Year Lumasiran Use in the DAILY-LUMA Cohort

Leclerc, A.-L. S., Cloarec, M., Knebelmann, B., Allard, L., Boyer, O., Cloarec, S., Dossier, C., Quintrec, M. L., Nobili, F., Stehlé, T., et al. (2024) · Kidney International Reports · doi:10.1016/j.ekir.2024.12.033 · Cited by 5 · see citing works

Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran

Titze-de-Almeida, S. S., Brandão, P. R. D. P., Faber, I., & Titze‐de‐Almeida, R. (2019) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-019-00434-w · Cited by 47 · see citing works

RNA-based therapies have their day

(2018) · EBioMedicine · doi:10.1016/j.ebiom.2018.08.025 · Cited by 2 · see citing works

Update on the clinical utility of an RNA interference-based treatment: focus on Patisiran

Rizk, M., & Tüzmen, Ş. (2017) · Pharmacogenomics and Personalized Medicine · doi:10.2147/pgpm.s87945 · Cited by 68 · see citing works

An evaluation of patisiran: a viable treatment option for transthyretin-related hereditary amyloidosis

Milani, P., Mussinelli, R., Perlini, S., Palladini, G. O., & Obici, L. P. (2019) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2019.1671352 · Cited by 11 · see citing works

Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis

Grogan, M. A., Sheikh, F. H., Sperry, B. W., Sweetser, M. T., Capocelli, K., Roblin, S., & Witteles, R. M. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102590 · Cited by 3 · see citing works