Results 191 to 200 of about 3,524,002 (263)

Hepatomegaly and Splenomegaly: An Approach to the Diagnosis of Lysosomal Storage Diseases. [PDF]

J Clin Med
Jerves Serrano T, Gold J, Cooper JA, Church HJ, Tylee KL, Wu HY, Kim SY, Stepien KM.   +7 more
europepmc   +1 more source

Clinical Experience With Eliglustat in Children With Gaucher Disease Type 1

American Journal of Medical Genetics Part A, EarlyView.
Erika R. Vucko, Allegra Quadri, Rachel E. Hickey, Carly A. Rasmussen, Monika Izdebski, Joel Charrow, Carlos E. Prada   +6 more
wiley   +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

Health care costs of home care enzyme replacement therapy for patients with lysosomal storage diseases in Germany. [PDF]

Orphanet J Rare Dis
Heinrich R, Claus F, Schoenfelder T.
europepmc   +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

The Liver and Lysosomal Storage Diseases: From Pathophysiology to Clinical Presentation, Diagnostics, and Treatment. [PDF]

Diagnostics (Basel)
Lipiński P, Tylki-Szymańska A.
europepmc   +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

A PIKfyve modulator combined with an integrated stress response inhibitor to treat lysosomal storage diseases. [PDF]

Proc Natl Acad Sci U S A
Hou WC, Massey LA, Rhoades D, Wu Y, Ren W, Frank C, Overkleeft HS, Kelly JW.   +7 more
europepmc   +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