Results 81 to 90 of about 173,792 (325)

Nanobody‐Decorated Lipid Nanoparticles for Enhanced mRNA Delivery to Tumors In Vivo

Advanced Healthcare Materials, EarlyView.
This study presents a targeted mRNA‐LNP platform engineered with anti‐PSMA nanobodies for prostate cancer therapy. Using different in vivo models, the authors demonstrate selective uptake and mRNA delivery in PSMA+ cancer cells. However, while targeting is enhanced, mRNA delivery efficacy is limited by tissue penetration, tumor necrosis, and immune ...
Pol Escudé Martinez de Castilla, Vincenzo Verdi, Willemijn de Voogt, Mariona Estapé Sentí, Arnold C. Koekman, Julian Rietveld, Sven van Kempen, Qiangbing Yang, Juliette van Merris, Guido Jenster, Martin E. van Royen, Marcel H. Fens, Sander A. A. Kooijmans, Wytske M. van Weerden, Guillaume van Niel, Pieter Vader, Raymond M. Schiffelers   +16 more
wiley   +1 more source

Exploring AAV‐Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential

Advanced Science, EarlyView.
Current preclinical studies of AAV‐mediated gene therapy explore different strategies based on the characteristics of inner ear diseases. For genetic hearing loss, approaches include the replacement of a “good gene,” removal of a “bad gene,” or direct correction of mutations through base editing.
Fan Wu, Wuhui He, Yun Xiao, Qiong Wu, Qing Zhang, Tiesong Zhang, Lei Xu, Xinyi Pang   +7 more
wiley   +1 more source

Controlling α-globin: a review of α-globin expression and its impact on β-thalassemia

Haematologica, 2008
Synthesis of α-globin and α-globin subunits of hemoglobin occurs at high levels during erythrocyte differentiation in a tightly controlled and coordinated fashion.
Hsiao Phin Joanna Voon, Jim Vadolas
doaj   +1 more source

Structural interpretation of the amino acid sequence of a second domain from the Artemia covalent polymer globin [PDF]

, 1990
Artemia has a complex extracellular hemoglobin of Mr 260,000 comprising two globin chains (Mr 130,000) each of which is a polymer of eight covalently linked domains of Mr 16,000.
Alard, Philippe, De Smet, Koen, Moens, Luc, Trotman, Clive, Van Beeumen, Jozef, Van de Peer, Yves, Van Hauwaert, Marie-Louise, Ver Donck, Kris, Wodak, Shoshana   +8 more
core   +1 more source

Lipoprotein lipase regulates hematopoietic stem progenitor cell maintenance through DHA supply. [PDF]

, 2018
Lipoprotein lipase (LPL) mediates hydrolysis of triglycerides (TGs) to supply free fatty acids (FFAs) to tissues. Here, we show that LPL activity is also required for hematopoietic stem progenitor cell (HSPC) maintenance.
Han, Tianxu, Kim, Jungsu, Klemke, Richard L, Liu, Chao, Miller, Yury I, Rana, Tariq M, Remaley, Alan T, Stachura, David L, Traver, David, Vaisman, Boris L, Wang, Huawei   +10 more
core   +2 more sources

A Machine‐Learning Approach Identifies Rejuvenating Interventions in the Human Brain

Advanced Science, EarlyView.
Rising life expectancy increases the incidence of age‐related neurodegeneration. Reverting brain aging through effective rejuvenation strategies constitutes a promising strategy to counteract these disorders and restore brain function. This study uses a brain‐specific transcriptional aging clock to screen over 43 000 perturbation profiles, identifying ...
Guillem Santamaria, Cristina Iglesias, Sascha Jung, Javier Arcos Hodar, Ruben Nogueiras, Antonio del Sol   +5 more
wiley   +1 more source

PRC1‐Mediated H2Aub Loop Formation and Function in Arabidopsis

Advanced Science, EarlyView.
In the Arabidopsis genome, H2Aub and H3K27me3 marks do not always co‐localize, and some regions are marked exclusively by H2Aub. By using Capture‐Hi‐C, it is found that H2Aub loops are preferentially established between two H2Aub enriched regions.
Lingxiao Luo, Minqi Yang, Zhihan Song, Yali Liu, Suxin Xiao, Dingyue Wang, Myriam Calonje, Hang He, Yue Zhou   +8 more
wiley   +1 more source

α-Globin as a molecular target in the treatment of β-thalassemia.

Blood, 2015
The thalassemias, together with sickle cell anemia and its variants, are the world's most common form of inherited anemia, and in economically undeveloped countries, they still account for tens of thousands of premature deaths every year.
S. Mettananda, R. Gibbons, D. Higgs
semanticscholar   +1 more source

Large‐Scale Production of Transfusion‐Ready Red Blood Cells From Induced Pluripotent Stem Cells

Advanced Science, EarlyView.
A scalable, dynamic culture system is developed to produce red blood cells from induced pluripotent stem cells. It achieves high yields with up to 70% enucleation, not previously shown in dynamic systems, overcoming key limitations and enabling large‐scale production.
Eszter Varga, Eelke Brandsma, Brenda E. Juarez‐Garza, Renuka P. E. Ramlal, Julien J. Karrich, Adrien Laurent, Athina Chavli, Ruthmila Paskel, Kerly Fu, Richard A. Flavell, Marieke von Lindern, Derk Amsen, Marieke E. Klijn, Emile van den Akker   +13 more
wiley   +1 more source

Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin

Nature Communications, 2015
Genetic disorders resulting from defects in the adult globin genes are among the most common inherited diseases. Symptoms worsen from birth as fetal γ-globin expression is silenced.
B. Wienert, Alister P. W. Funnell, Laura J. Norton, Richard C. M. Pearson, Lorna Wilkinson-White, Krystal L. Lester, J. Vadolas, M. Porteus, J. Matthews, K. Quinlan, M. Crossley   +10 more
semanticscholar   +1 more source