Results 141 to 150 of about 40,385 (227)

Novel Fused Pyrazole Amide Analogs as Glucosylceramide Synthase Inhibitors for Treating Lysosomal Storage Diseases, Neurodegenerative Diseases, Cystic Diseases, and Cancer. [PDF]

ACS Med Chem Lett, 2023
Sabnis RW.
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

Human, economic, and social impact of lysosomal storage diseases. [PDF]

Orphanet J Rare Dis
Brignani E, de Castro-López MJ, Gonzalez-Meneses A, Morales M, Pintos-Morell G, Poveda JL, Vilalta I, Cruz J, Ballester-Lozano GF, David de Paz H.   +9 more
europepmc   +1 more source

Nonimmune Hydrops Fetalis and Lysosomal Storage Diseases

Pediatrics and Neonatology, 2013
Carlo Bellini
doaj   +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

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

Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]

EBioMedicine
eBioMedicine.
europepmc   +1 more source

Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches. [PDF]

Front Mol Neurosci, 2022
Flunkert S, Hutter-Paier B, Sun Y, Kehr J.   +3 more
europepmc   +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

Japanese experience of newborn screening for lysosomal storage diseases and adrenoleukodystrophy. [PDF]

Orphanet J Rare Dis
Onuki T, Tajika M, Sugiyama Y, Shimura M, Ichimoto K, Tanaka T, Nyuzuki H, Kosuga M, Migita O, Ito T, Sasai H, Bo R, Hamada J, Hamazaki T, Sakai N, Inoue T, Nakamura K, Okuyama T, Murayama K.   +18 more
europepmc   +1 more source