Results 131 to 140 of about 25,002 (280)

Reactivation of mTOR signaling slows neurodegeneration in a lysosomal sphingolipid storage disease

Neurobiology of Disease
Sandhoff disease, a lysosomal storage disorder, is caused by pathogenic variants in the HEXB gene, resulting in the loss of β-hexosaminidase activity and accumulation of sphingolipids including GM2 ganglioside.
Hongling Zhu, Y. Terry Lee, Colleen Byrnes, Jabili Angina, Danielle A. Springer, Galina Tuymetova, Mari Kono, Cynthia J. Tifft, Richard L. Proia   +8 more
doaj   +1 more source

Harnessing ferroptosis from multilayer defense networks to nanoplatforms for specific cancer therapy

BMEMat, EarlyView.
Nanomaterials target metabolically‐regulated ferroptosis for cancer therapy. Iron‐based or alternative nanoplatforms integrate ferroptosis with chemotherapy, immunotherapy, or radiotherapy. They enable stimulus‐responsive therapies (photothermal, photodynamic, sonodynamic) activated by near‐infrared, light, or ultrasound, achieving potent synergistic ...
Xinyue Xu, Xuejian Wang, Wenyan Jiang, Wan Hairul Anuar Kamaruddin, Chunyan Fang, Pengfei Zhang   +5 more
wiley   +1 more source

Transfersomes with core and surface‐loaded NF‐κB p65 siRNA for enhanced transdermal transfection and effective treatment of psoriasis

BMEMat, EarlyView.
TCPL, a transfersome with Tween 80 and PEI, enables efficient transdermal siNF‐κB delivery, resolving PEI's toxicity issue. It promotes lysosomal escape and effective transfection, silences NF‐κB, modulates immunity, and inhibits angiogenesis, alleviating psoriasis. With good biocompatibility, TCPL holds strong clinical translation potential. Schematic
Hui Xing, Yuhui Yang, Ziyi Zhao, Kai Lv, Yiran Shao, Zhi Zhang, Dong Ma   +6 more
wiley   +1 more source

Integrated spherical nucleic acid nanoreactor mobilizes targeted PD‐L1 proteolysis and cuproptosis for robust breast cancer immunotherapy

BMEMat, EarlyView.
This study provides an integrated spherical nucleic acid nanoreactor (denoted as AMCuD) for targeted PD‐L1 degradation and chemo‐dynamically enhanced cuproptosis to synergically elicit robust immunotherapy against the triple‐negative breast cancer (TNBC) growth, recurrence and metastasis.
Ningxi Li, Honglin Huang, Qianyi Zeng, Li Zeng, Ziman Yang, Jinyang Shen, Shun Li, Hong Yang, Yiyao Liu, Chunhui Wu   +9 more
wiley   +1 more source

Treatment of lysosomal storage disorders: successes and challenges

, 2014
Treatment options for a number of lysosomal storage disorders have rapidly expanded and currently include enzyme replacement therapy, substrate reduction, chaperone treatment, hematopoietic stem cell transplantation, and gene-therapy.
Carla E. M. Hollak, Frits A. Wijburg, Hollak, Carla E. M., Wijburg, Frits A.   +3 more
core   +1 more source

Tackling cancer stemness with nanotechnology in the era of precision medicine

BMEMat, EarlyView.
Precise customization of nanoparticles (NPs) enables active targeting of cancer stem cells (CSCs), thereby improving drug delivery and therapeutic efficacy. NP‐based probing enhances CSC detection through imaging and liquid biopsy, whereas diverse therapeutic payloads improve therapeutic outcomes.
Shaolei Guo, Shun Yao, Lihui Meng, Chunyuan Chen, Yuechu Lucinda Lu, Rong Lu, Kashif Rafiq Zahid, Umar Raza, Qingzhi Wu, Wenli Chen   +9 more
wiley   +1 more source

First National Expanded Genomic Newborn Screening Program in Qatar; A Pilot Study, Doha‐Heidelberg Collaboration

American Journal of Medical Genetics Part A, EarlyView.
Reem Alsulaiman, Sarah Okashah, Radja Messai Badji, Fatemeh Abbaszadeh, Yaser Alsarraj, Rehab Ali Abdulrahman, Noora Shahbeck, Thalib Moideen, Reema El Hassan, Fatima Al‐Hamaydeh, Einas Alkuwari, Said Ismail, Hilal Al‐ Rifai, Ghassan Abdoh, Christian P. Schaaf, Georg Hoffmann, Saad Al‐Kaabi, Wadha Al‐Muftah, Tawfeg Ben‐Omran   +18 more
wiley   +1 more source

Status and future of recombinant adeno‐associated virus vector manufacturing

Biotechnology Progress, EarlyView.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

Organelle‐Resolved Tetrazine‐trans‐Cyclooctene Click Chemistry for Cargo Delivery and Release

Chemistry – A European Journal, EarlyView.
Bioorthogonal click chemistry tools provide a means for specific intracellular conjugation of molecules. In this study, we used reactive tetrazine (Tz) and TCO moieties for labeling of organelles and organelle‐specific delivery and activation of doxorubicin prodrugs.
Oleh Durydivka, Marek Chovanec, Rastislav Dzijak, Michal Rahm, Jachym Hrusak, Paul E. Reyes‐Gutierrez, Milan Vrabel   +6 more
wiley   +1 more source

Pharmacological small molecules for the treatment of lysosomal storage disorders

, 2010
Importance of the field: Inherited lysosomal storage diseases often cause severe disability and have a devastating effect on quality of life. Enzyme replacement therapy (ERT) forms a cornerstone in the treatment of lysosomal enzyme deficiencies. Although
Aerts, J. M. F. G., BE Smid, Smid, B. E., RG Boot, Linthorst, G. E., Hollak, C. E. M., GE Linthorst, JMFG Aerts, Boot, R. G., CEM Hollak   +9 more
core   +1 more source