Engineering precision nanoparticles for drug delivery

MJ Mitchell, MM Billingsley, RM Haley… - Nature reviews drug …, 2021 - nature.com
In recent years, the development of nanoparticles has expanded into a broad range of
clinical applications. Nanoparticles have been developed to overcome the limitations of free …

Strategies for non-viral vectors targeting organs beyond the liver

J Kim, Y Eygeris, RC Ryals, A Jozić, G Sahay - Nature nanotechnology, 2024 - nature.com
In recent years, nanoparticles have evolved to a clinical modality to deliver diverse nucleic
acids. Rising interest in nanomedicines comes from proven safety and efficacy profiles …

Regulating tumor suppressor genes: post-translational modifications

L Chen, S Liu, Y Tao - Signal transduction and targeted therapy, 2020 - nature.com
Tumor suppressor genes cooperate with each other in tumors. Three important tumor
suppressor proteins, retinoblastoma (Rb), p53, phosphatase, and tensin homolog deleted …

Ionizable lipid nanoparticles for in utero mRNA delivery

RS Riley, MV Kashyap, MM Billingsley, B White… - Science …, 2021 - science.org
Clinical advances enable the prenatal diagnosis of genetic diseases that are candidates for
gene and enzyme therapies such as messenger RNA (mRNA)–mediated protein …

Peptide nucleic acid (PNA) and its applications in chemical biology, diagnostics, and therapeutics

J Saarbach, PM Sabale, N Winssinger - Current opinion in chemical …, 2019 - Elsevier
Peptide nucleic acid (PNA) stands as one of the most successful artificial oligonucleotide
mimetics. Salient features include the stability of hybridization complexes (either as …

PTEN-opathies: from biological insights to evidence-based precision medicine

L Yehia, J Ngeow, C Eng - The Journal of clinical …, 2019 - Am Soc Clin Investig
The tumor suppressor phosphatase and tensin homolog (PTEN) classically counteracts the
PI3K/AKT/mTOR signaling cascade. Germline pathogenic PTEN mutations cause PTEN …

Spotlight on genetic kidney diseases: A call for drug delivery and nanomedicine solutions

N Trac, A Ashraf, J Giblin, S Prakash, S Mitragotri… - ACS …, 2023 - ACS Publications
Nanoparticles as drug delivery carriers have benefited diseases, including cancer, since the
1990s, and more recently, their promise to quickly and efficiently be mobilized to fight …

Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery

KL Swingle, MM Billingsley, SK Bose, B White… - Journal of Controlled …, 2022 - Elsevier
Congenital disorders resulting in pathological protein deficiencies are most often treated
postnatally with protein or enzyme replacement therapies. However, treatment of these …

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

E Magrin, A Miccio, M Cavazzana - Blood, The Journal of the …, 2019 - ashpublications.org
Abstract β-Thalassemia and sickle cell disease (SCD) are the most prevalent monogenic
diseases. These disorders are caused by quantitative or qualitative defects in the production …

The challenges and opportunities in the development of MicroRNA therapeutics: a multidisciplinary viewpoint

MY Momin, RR Gaddam, M Kravitz, A Gupta, A Vikram - Cells, 2021 - mdpi.com
microRNAs (miRs) are emerging as attractive therapeutic targets because of their small size,
specific targetability, and critical role in disease pathogenesis. However,< 20 miR targeting …