Results 51 to 60 of about 4,682 (148)

Glikoznanost - nova smjernica u suvremenom dizajniranju lijekova [PDF]

, 2006
Glycans are the most abundant and most diverse biopolymers in nature. Because of their highly specific interactions with physiological receptors, they participate in many crucial biological processes.
GORDAN LAUC, JERKA DUMIC, MIRNA FLÖGEL, OLGA GORNIK   +3 more
core   +1 more source

CRISPLD2 Attenuates Intervertebral Disc Degeneration by Suppressing Oxidative Stress‐Induced Ferroptosis through the miR‐548I‐IL17A Axis

Advanced Science, Volume 13, Issue 16, 18 March 2026.
This study identifies CRISPLD2 as a key protector against IVDD. By regulating ferroptosis through the CRISPLD2–miR‐548I–IL17A axis, CRISPLD2 maintains NPCs homeostasis and reduces oxidative stress. Restoring CRISPLD2 expression effectively alleviates disc degeneration and highlights a promising therapeutic strategy for discogenic low back pain ...
Yangyang Shi, Fudong Li, Tianyi Zhao, Kaiqiang Sun, Danying Zhang, Chen Yan, Ximing Xu, Jingchuang Sun, Xulin Hu, Xin Yong, Jiangang Shi, Yuan Wang   +11 more
wiley   +1 more source

Molecular Pharming: Advances, Applications, and Future Prospects in Biotechnology and Medicine

Engineering in Life Sciences, Volume 26, Issue 3, March 2026.
ABSTRACT Genetically engineered plants incorporate the use of a novel bioreactor known as molecular pharming, which has a transformative view on the pharmaceutical industry. The technique enables mass production, at a low cost, and reproducibly of a large number of different protein‐based drugs, vaccines, and industrial enzymes. This review‐based study
Md. Hridoy Ahmed, Md. Mustak Khan, Shishir Dutta, Md. Foyzur Rahman, Mohammad Shariful Islam, Md. Najmul Hosen, Md. Saad Hossain, Md. Aftabur Rahman, Md. Sadman Hasan Sahil, Tanjuma Tasnim Hira, Ifthesum, Ashikur Rahaman, Md. Afser Rabbi, Laila Khaleda   +13 more
wiley   +1 more source

Linear cyclodextrin polymer prodrugs as novel yherapeutics for Niemann-Pick type C1 disorder [PDF]

, 2018
Niemann-Pick Type C1 disorder (NPC) is a rare lysosomal storage disease characterized by the accumulation of cholesterol in lysosomes. NPC has no FDA approved treatments yet, however 2-hydroxypropyl-β-cyclodextrin (HPβCD) has shown efficacy for treating ...
Aditya Kulkarni, Alberto Macone, Atul Dolas, Jessica Dragotto, Maria Teresa Fiorenza, Paola Caporali, Ramesh Jayaraman, Sandeep Goyal, Soniya Johny   +8 more
core   +1 more source

Synuclein Disorder‐Related Genetic Determinants of Mild Behavioural Impairment in a Pre‐Clinical Community Cohort

International Journal of Geriatric Psychiatry, Volume 41, Issue 3, March 2026.
ABSTRACT Background The GBA variant confers increased risk of synuclein disorders but it is unclear what impact it has in pre‐clinical groups. This study aimed to identify early psychiatric and cognitive manifestations amongst pre‐clinical GBA carriers in a community cohort.
Millie Sander‐Long, Byron Creese, Anne Corbett, Ivana Rosenzweig, Jeffrey Cummings, Clive Ballard   +5 more
wiley   +1 more source

High frequency of mutation G377S in Brazilian type 3 Gaucher disease patients [PDF]

, 2006
Gaucher disease (GD), the most prevalent lysosome storage disorder, presents an autosomal recessive mode of inheritance. It is a paradigm for therapeutic intervention in medical genetics due to the existence of effective enzyme replacement therapy.
Aranda, Paulo Cesar, Araujo, Fernando T., Cravo, Renata, Fox, Deborah Catherine, Martins, Ana Maria [UNIFESP], Micheletti, Cecília [UNIFESP], Nonino, Alexandre, Pereira, Lygia da Veiga, Rozenberg, Roberto, Sobreira, Elisa   +9 more
core   +1 more source

Medicine Development and Access for Rare Diseases: Can We Do Better?

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Recent advances in molecular biology and genomics have significantly enhanced our understanding of rare diseases. While enabling the development of highly targeted therapies, it also leads to complexity in the development, regulation, and accessibility of orphan medicines.
Carla E. M. Hollak, Noa Rosenberg, Colinda Post, Nina Stolwijk, Evert Manders, Daphne Schoenmakers, Bart Penninx, Niels Reijnhout, Kathelijn Verdeyen, Roel Jonker, Annet M. Bosch, Mirjam Langeveld, Vincent van der Wel, Saco de Visser, Sibren van den Berg   +14 more
wiley   +1 more source

Splenic infarction: an update on William Osler\u27s observations. [PDF]

, 2010
BACKGROUND: Osler taught that splenic infarction presents with left upper abdominal quadrant pain, tenderness and swelling accompanied by a peritoneal friction rub.
Aharoni, MD, Dvora, Berlowitz, MB BCh, Daniel, Breuer, MD, Gabriel S, Hain, MD, Daniel, Lawrence, MA MBBS MRCP, Yaacov R, Pokroy, MB BCh, Russell   +5 more
core   +1 more source

Pleiotropy as the Mechanism for Evolving Novelty: Same Signal, Different Result. [PDF]

, 2015
In contrast to the probabilistic way of thinking about pleiotropy as the random expression of a single gene that generates two or more distinct phenotypic traits, it is actually a deterministic consequence of the evolution of complex physiology from the ...
Torday, John S
core   +2 more sources

RNA‐Based Therapies for Inherited Metabolic Disorders

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri, Sonam Gurung, Roopkatha Ghosh, Philippa B. Mills, Julien Baruteau, Haiyan Zhou   +5 more
wiley   +1 more source