Results 61 to 70 of about 6,119 (148)

Acid Sphingomyelinase-deficient Macrophages Have Defective Cholesterol Trafficking and Efflux [PDF]

Journal of Biological Chemistry, 2001
Cholesterol efflux from macrophage foam cells, a key step in reverse cholesterol transport, requires trafficking of cholesterol from intracellular sites to the plasma membrane. Sphingomyelin is a cholesterol-binding molecule that transiently exists with cholesterol in endosomes and lysosomes but is rapidly hydrolyzed by lysosomal sphingomyelinase (L ...
A R, Leventhal, W, Chen, A R, Tall, I, Tabas   +3 more
openaire   +2 more sources

The Liver and Lysosomal Storage Diseases: From Pathophysiology to Clinical Presentation, Diagnostics, and Treatment

Diagnostics
The liver, given its role as the central metabolic organ, is involved in many inherited metabolic disorders, including lysosomal storage diseases (LSDs).
Patryk Lipiński, Anna Tylki-Szymańska
doaj   +1 more source

Purification of Advanced Therapeutics by Flow‐Through Pseudo‐Affinity Chromatography: Bi‐Specific mABs, Fc Fusion Proteins, and Adeno‐Associated Viruses

Biotechnology and Bioengineering, Volume 123, Issue 11, Page 3079-3105, November 2026.
ABSTRACT Modern biopharmaceutical manufacturing requires purification platforms capable of processing structurally and functionally diverse products while addressing the challenge of removing persistent and high‐risk host cell proteins (HCPs).
Wenning Chu, Eduardo Barbieri, Arianna Minzoni, Shriarjun Shastry, Jacob P. LeBarre, Qingbo Shu, Taufika Williams, Xuanwen Li, Divya Chandra, Xuankuo Xu, Jerome Bill, David Roush, Joseph Taris, Patrick Gilbert, Andre Kiesewetter, Oliver Rammo, Michael Schulte, Christopher Major, Trina Mouchahoir, Steven Cramer, Cristiana Boi, Ruben G. Carbonell, Abraham Lenhoff, Stefano Menegatti   +23 more
wiley   +1 more source

Hepatic HIF2α Modulates Extra‐Hepatic Disease‐Associated Phenotypes During Metabolic Dysfunction‐Associated Steatotic Liver Disease

Comprehensive Physiology, Volume 16, Issue 5, October 2026.
High fat, high fructose, high cholesterol “GAN” diet feeding in mice induces MASLD, cardiac dysfunction, loss of lean mass, and hyperinsulinaemia. Hepatocyte‐specific deletion of HIF2α also causes cardiac dysfunction and loss of lean mass but prevents GAN induced hyperinsulinaemia and cardiac sympathetic dominance without preventing MASLD.
Lorenz M. W. Holzner, Rosa M. Korpershoek, Youguo Niu, Anna Cochrane, Paula M. Darwin, Julia Babuta, Areesha Nazeer, Cecilia Castro, Alice P. Sowton, Alice E. Knapton, Benjamin D. Thackray, Julian L. Griffin, Zoe Hall, Dino A. Giussani, Rob C. I. Wüst, Andrew J. Murray   +15 more
wiley   +1 more source

Heparin Can Augment Extracellular Vesicle Yield by Inhibiting Reuptake in Donor Cells During Cell Propagation

Journal of Extracellular Vesicles, Volume 15, Issue 10, October 2026.
Heparin augments EV yield in culture by inhibiting EV binding to cell‐surface HSPG and masking EV surface. Preventing autocrine and homotypic paracrine EV uptake during cell culture results in EV accumulation and higher yield. Heparin improved EV recovery after storage in NaCl but not in complex media or protein‐rich buffer(Please see Figure 9 for ...
André Cronemberger Andrade, Rodolphe Poupardin, Martin Wolf, Liliia Paniushkina, Karim Selmi, Deepika Kundully Suresh, Nicole Maeding, Heide‐Marie Binder, Janine Zwicklhuber, Sarah Hochmann, André Görgens, Samir El Andaloussi, Katharina Schallmoser, Dirk Strunk   +13 more
wiley   +1 more source

Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results

Orphanet Journal of Rare Diseases, 2022
Background Olipudase alfa is a recombinant human acid sphingomyelinase (ASM) enzyme replacement therapy (ERT) for non-central-nervous-system manifestations of acid sphingomyelinase deficiency (ASMD).
George A. Diaz, Roberto Giugliani, Nathalie Guffon, Simon A. Jones, Eugen Mengel, Maurizio Scarpa, Peter Witters, Abhimanyu Yarramaneni, Jing Li, Nicole M. Armstrong, Yong Kim, Catherine Ortemann-Renon, Monica Kumar   +12 more
doaj   +1 more source

Senescent Cell‐Derived Extracellular Vesicles in Therapy‐Induced Senescence: Immune Remodelling and Emerging Links to Mitochondrial Stress in Cancer

Journal of Extracellular Biology, Volume 5, Issue 10, October 2026.
ABSTRACT Cancer therapy can induce cellular senescence and remodel the tumour microenvironment through the senescence‐associated secretory phenotype (SASP), including its membrane‐enclosed component, senescent cell‐derived extracellular vesicles (senEVs). This review summarizes senEV biogenesis, cargo‐dependent functions, and immune‐remodelling effects
Yongrui Qian, Zirui Weng, Na Li, Yun Zhou, Hailong Pei   +4 more
wiley   +1 more source

Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial

Orphanet Journal of Rare Diseases, 2023
Background Olipudase alfa is a recombinant human acid sphingomyelinase enzyme replacement therapy for non-central-nervous-system manifestations of acid sphingomyelinase deficiency (ASMD). The ASCEND randomized placebo-controlled trial in adults with ASMD
Melissa P. Wasserstein, Robin Lachmann, Carla Hollak, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto Bernardo Guelbert, Julia B. Hennermann, Takayuki Ikezoe, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Abhimanyu Yarramaneni, Nicole M. Armstrong, Yong Kim, Monica Kumar   +20 more
doaj   +1 more source

Extracellular Vesicles: Classification, Biological Functions, Diseases, and Therapeutic Opportunities

MedComm, Volume 7, Issue 10, October 2026.
• EV content maintains cellular homeostasis and is involved in several disease onset and progression. • miRNA export in EVs depends on: RNA‐binding proteins recognizing motif sequences and on epitranscriptomic modifications. • The disclosure of delivery mechanisms is crucial for developing personalized EV‐based therapeutic tools. ABSTRACT Extracellular
Sabrina Garbo, Francesco Marocco, Clemens Zwergel, Marco Tripodi, Cecilia Battistelli   +4 more
wiley   +1 more source

An Early-Onset Neuronopathic Form of Acid Sphingomyelinase Deficiency: A SMPD1 p.C133Y Mutation in the Saposin Domain of Acid Sphingomyelinase.

The Tohoku journal of experimental medicine, 2020
Acid sphingomyelinase (ASM) is a lysosomal hydrolase that degrades sphingomyelin into ceramide and phosphocholine. Recent crystallographic studies revealed the functional role of the N-terminal ASM saposin domain. ASM deficiency due to mutations in the ASM-encoding sphingomyelin phosphodiesterase 1 (SMPD1) gene causes an autosomal recessive ...
Ota, Shozo, Noguchi, Atsuko, Kondo, Daiki, Nakajima, Yoko, Ito, Tetsuya, Arai, Hirokazu, Takahashi, Tsutomu   +6 more
openaire   +2 more sources