Results 151 to 160 of about 36,163 (246)

Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases. [PDF]

Am J Med Genet C Semin Med Genet
Borges B, Canepa E, Chang IJ, Herzeg A, Lianoglou B, Kishnani PS, Harmatz P, MacKenzie TC, Cohen JL.   +8 more
europepmc   +1 more source

Carrier‐free self‐assembled nanomedicine for combination‐therapy of acute myeloid leukemia

BMEMat, EarlyView.
CPDS combines drugs with three different mechanisms of action to achieve a multi‐mechanism combination therapy for AML by directly killing tumor cells and activating anti‐tumor immunity. Abstract As the main acute myeloid leukemia (AML) clinical treatment, the chemotherapy alone cannot meet the clinical therapeutic needs due to the high heterogeneity ...
Meihong Chai, Rui Zhang, Haiyin Yang, Jiyue Xing, Tianyuan Hu, Mengxi Zhu, Rui Li, Xiaoli Chen, Beibei Zhao, Yang Lan, Stefan Barth, Yuanyu Huang, Li Zhang, Mengjie Zhang, Huining He   +14 more
wiley   +1 more source

Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach. [PDF]

JIMD Rep
Corazolla EM, Langeveld M, Brands MMMG, Sjouke B, Hollak CEM.   +4 more
europepmc   +1 more source

Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier. [PDF]

Mol Ther, 2023
Critchley BJ, Gaspar HB, Benedetti S.
europepmc   +1 more source

Dynamics of natural killer cell function upon recurrent stimulation

Biotechnology Progress, EarlyView.
Weekly stimulation of peripheral blood‐derived NK cells with engineered K562 feeder cells resulted in two distinct growth phases, shifting around Day 21–26. Cells in growth phase 2 exhibited slower growth, reduced metabolic activity, and lower cytotoxicity than cells from growth phase 1, which has implications for NK cell biomanufacturing.
Jennifer One, Janani Narayan, Frank Cichocki, Wei‐Shou Hu, Samira M. Azarin   +4 more
wiley   +1 more source

c-Abl/TFEB Pathway Activation as a Common Pathogenic Mechanism in Lysosomal Storage Diseases: Therapeutic Potential of c-Abl Inhibitors. [PDF]

Antioxidants (Basel)
Guerra MV, Castro J, Moreno A, Balboa E, Marugan JJ, Alvarez AR, Zanlungo S.   +6 more
europepmc   +1 more source

The Role of the miR-17-92 Cluster in Autophagy and Atherosclerosis Supports Its Link to Lysosomal Storage Diseases. [PDF]

Cells, 2022
Ortuño-Sahagún D, Enterría-Rosales J, Izquierdo V, Griñán-Ferré C, Pallàs M, González-Castillo C.   +5 more
europepmc   +1 more source

Boron Neutron Capture Therapy at a Crossroads: Translational Gap and Emerging Delivery Agents

Chemistry – A European Journal, EarlyView.
This review surveys recent advances in boron delivery agents for BNCT, emphasizing the shift from classical small molecules to multifunctional nanocarriers and theranostic systems. By integrating targeting, imaging, and therapy, next‐generation boron compounds aim to bridge the gap between (bio)chemical innovation and clinical translation.
Christoph Selg, Evamarie Hey‐Hawkins
wiley   +1 more source

Lysosomal storage diseases in North America: a comprehensive review of enzyme therapies and unmet needs. [PDF]

Ther Adv Rare Dis
Bennett LL.
europepmc   +1 more source

Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases. [PDF]

Mol Ther, 2023
Heller G, Bradbury AM, Sands MS, Bongarzone ER.   +3 more
europepmc   +1 more source