Results 91 to 100 of about 172,174 (293)

ApoE gene therapy: an overview and update

, 2005
Atherosclerosis remains the leading cause of death in industrialized societies. Apolipoprotein E (ApoE) is an attractive candidate to treat hypercholesterolemia and coronary heart disease, as it is a circulating protein with pleiotropic ...
Owen, J.S.
core  

Targeting Amyotrophic Lateral Sclerosis with Gene Therapy: From Silencing Genes to Enhancing Neuroprotection [PDF]

Elivery routes; Gene augmentation; Gene silencingVies d'administració; Augment gènic; Silenciament gènicVías de administración; Aumento génico; Silenciamiento génicoGene therapy is emerging as a transformative approach for treating amyotrophic lateral ...
Verdés-Franquesa, Sergi, Bosch, Assumpció, Navarro, Xavier   +2 more
core   +1 more source

Gene Therapy of Adrenomyeloneuropathy: Challenges, Target Cells, and Prospectives

Biomedicines
Gene replacement using adeno-associated viral (AAV) vectors has become a major therapeutic avenue for neurodegenerative diseases (NDD). In single-gene diseases with loss-of-function mutations, the objective of gene therapy is to express therapeutic ...
Pierre Bougnères, Catherine Le Stunff, Romina Aron Badin   +2 more
doaj   +1 more source

Proteasome inhibitor, ixazomib prevents topoisomerase‐I degradation and reverses irinotecan resistance in colorectal cancer

Molecular Oncology, EarlyView.
Ixazomib inhibits proteasome‐mediated degradation of topoisomerase I induced by irinotecan, thereby restoring drug sensitivity and promoting tumor cell death in colorectal cancer. Irinotecan, a topoisomerase I (topoI) inhibitor, is widely used for colorectal cancer, but resistance remains a major clinical challenge.
Yuho Ebata, Koji Ando, Hirofumi Hasuda, Koshi Mimori, Elizabeth C. Unan, Siddhartha Pulukuri, Aahana Tiku, Allison Berger, Eiji Oki, Ajit Bharti, Tomoharu Yoshizumi   +10 more
wiley   +1 more source

mRNA in the Context of Protein Replacement Therapy

, 2023
Protein replacement therapy is an umbrella term used for medical treatments that aim to substitute or replenish specific protein deficiencies that result either from the protein being absent or non-functional due to mutations in affected patients ...
Theofanis Vavilis, Ioannis Dardalas, Sachinidis, Athanasios, Ainatzoglou, Alexandra, Eleni Stamoula, Athanasios Sachinidis, Lamprinou, Malamatenia, Vizirianakis, Ioannis S., Alexandra Ainatzoglou, Ioannis S. Vizirianakis, Vavilis, Theofanis, Dardalas, Ioannis, Malamatenia Lamprinou, Stamoula, Eleni   +13 more
core   +1 more source

DNA methylation and expression of MAPRE3 affect overall survival of early‐stage non‐small cell lung cancer patients

Molecular Oncology, EarlyView.
Both cg12821679MAPRE3 methylation and MAPRE3 expression are significantly associated with overall survival (OS) of non‐small cell lung cancer. Meanwhile, MAPRE3 expression significantly modified the effect of smoking cessation on OS. Smoking cessation benefits OS merely for patients with high MAPRE3 expression.
Chao Chen, Jiancheng Cheng, Ruili Hou, Xiaoyuan Zheng, Li Su, Maria Moksnes Bjaanæs, Anna Karlsson, Maria Planck, Johan Staaf, Åslaug Helland, Manel Esteller, David C. Christiani, Feng Chen, Xiaofei Cao, Ruyang Zhang   +14 more
wiley   +1 more source

Psychological well-being on thyroid hormone replacement [PDF]

Despite 100 years after the discovery of thyroxine, controversy still exists regarding optimal thyroid hormone replacement therapy. Several anecdotal reports suggest that thyroxine alone therapy does not normalise psychological wellbeing.
Saravanan, Ponnusamy
core  

Perinatal Gene Transfer to the Liver

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Ward, N.J., Buckley, S.M.K., Ward, NJ, Chan, J.K.Y., Waddington, S.N., Rahim, A.A., Waddington, SN, Rahim, AA, Chan, JK, Mckay, T.R., Howe, S.J., Howe, SJ, McKay, TR   +13 more
core   +1 more source

Evaluation of MYBPC3 trans-splicing and gene replacement as therapeutic options in human iPSC-derived cardiomyocytes

, 2022
475486Gene therapy is a promising option for severe forms of genetic diseases. We previously provided evidence for the feasibility of trans-splicing, exon skipping, and gene replacement in a mouse model of hypertrophic cardiomyopathy (HCM) carrying a ...
Mearini, G., Pless, O., Redwood, C., Shibamiya, A., Eschenhagen, T., Flenner, F., Müller, O.J., Prondzynski, M., Münch, J., Hansen, A., Patten, M., Laufer, S.D., Carrier, L., Krämer, E.   +13 more
core   +1 more source

Clinical performance of the urine‐based TERT promoter AbsoluteQ Digital PCR for non‐invasive detection of bladder cancer

Molecular Oncology, EarlyView.
A urine‐based digital PCR assay targeting two hotspot TERT promoter variants detected bladder cancer with high sensitivity and no false positives in this case–control cohort. The streamlined AbsoluteQ workflow outperformed Sanger sequencing and supports non‐invasive molecular testing for bladder cancer detection.
Anna Nykel, Izabela Kubiak, Lena Rutkowska, Żaneta Kasprzyk, Łukasz Kępczyński, Michał Bednarek, Dariusz Sobieraj, Jacek Wilkosz, Piotr Kania, Adam Jędrzejczyk, Bogdan Kałużewski, Agnieszka Gach, Tadeusz Kałużewski   +12 more
wiley   +1 more source