Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

MK Tsoumpra, S Fukumoto, T Matsumoto, S Takeda… - …, 2019 - thelancet.com
Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive
muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense …

Bone-targeted nanoplatform combining zoledronate and photothermal therapy to treat breast cancer bone metastasis

W Sun, K Ge, Y Jin, Y Han, H Zhang, G Zhou, X Yang… - ACS …, 2019 - ACS Publications
Bone metastasis, a clinical complication of patients with advanced breast cancer, seriously
reduces the quality of life. To avoid destruction of the bone matrix, current treatments focus …

Pharmacological modulation of mitochondrial ion channels

L Leanza, V Checchetto, L Biasutto… - British journal of …, 2019 - Wiley Online Library
The field of mitochondrial ion channels has undergone a rapid development during the last
three decades, due to the molecular identification of some of the channels residing in the …

Tunneling nanotubular expressways for ultrafast and accurate M1 macrophage delivery of anticancer drugs to metastatic ovarian carcinoma

L Guo, Y Zhang, Z Yang, H Peng, R Wei, C Wang… - ACS …, 2019 - ACS Publications
It is extremely difficult for cancer chemotherapy to control the peritoneal metastasis of
advanced ovarian carcinoma given its inability to target disseminated tumors and the severe …

[HTML][HTML] Antisense oligonucleotides for the treatment of cardiomyopathy in Duchenne muscular dystrophy

Q Nguyen, T Yokota - American journal of translational research, 2019 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is an X-linked recessive fatal neuromuscular disorder
characterized by progressive muscle degeneration which affects one in 3500-5000 males …

Nanomedicines for the Delivery of Biologics

J Wahlich, A Desai, F Greco, K Hill, AT Jones, RJ Mrsny… - 2019 - mdpi.com
A special symposium of the Academy of Pharmaceutical Sciences Nanomedicines Focus
Group reviewed the current status of the use of nanomedicines for the delivery of biologics …

Identification of novel medulloblastoma cell-targeting peptides for use in selective chemotherapy drug delivery

KC Tjandra, N McCarthy, L Yang, AJ Laos… - Journal of medicinal …, 2019 - ACS Publications
Medulloblastoma is a malignant brain tumor diagnosed in children. Chemotherapy has
improved survival rates to approximately 70%; however, children are often left with long-term …

Paclitaxel-loaded folate-coated pH-sensitive liposomes enhance cellular uptake and antitumor activity

LOF Monteiro, RS Fernandes, L Castro… - Molecular …, 2019 - ACS Publications
Paclitaxel (PTX) is a microtubule-stabilizing agent widely used to treat breast cancer.
Nevertheless, the low solubility of the drug and the side effects of commercial formulations …

How to evaluate the cellular uptake of CPPs with fluorescence techniques: Dissecting methodological pitfalls associated to tryptophan-rich peptides

Q Seisel, F Pelletier, S Deshayes… - Biochimica et Biophysica …, 2019 - Elsevier
Cell-penetrating peptides (CPP) are broadly recognized as efficient non-viral vectors for the
internalization of compounds such as peptides, oligonucleotides or proteins. Characterizing …

Towards an understanding of the self-assembly and applications of phospholipid-containing nanoparticles

SCL Hall - 2019 - etheses.bham.ac.uk
Polymer-stabilized phospholipid nanodiscs are nanoscale, discoidal assemblies containing
a central core of lipid bilayer stabilized in aqueous solution by an amphipathic copolymer …