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Gene Therapy: Towards a New Era of Medicine.
AAPS PharmSciTech
December 2024
Chitkara College of Pharmacy, Chitkara University, Rajpura, Punjab, India.
Over the past years, many significant advances have been made in the field of gene therapy and shown promising results in clinical trials conducted. Gene therapy aims at modifying or replacing a defective, inefficient, or nonfunctional gene with a healthy, functional gene by administration of genome material into the cell to cure genetic diseases. Various methods have been devised to do this by using several viral and non-viral vectors which are either administered by in vivo or ex vivo technique.
View Article and Find Full Text PDFReduction of Ovarian Cysts After Endoscopic Surgery for Follicle-Stimulating Hormone-Producing Pituitary Adenoma.
JCEM Case Rep
December 2024
Department of Diabetes and Endocrinology, University of Yamanashi Hospital, Yamanashi 4093898, Japan.
A 49-year-old woman presented with irregular menstrual bleeding, elevated estradiol (E2) (665 pg/mL [2441.21 pmol/L]) (reference range [RR]: menstrual period [MP] 20-50 pg/mL; 73.42-183.
View Article and Find Full Text PDFA phase I study assessing the safety and tolerability of SPL84, an inhaled antisense oligonucleotide for treatment of cystic fibrosis patients with the 3849 +10kb C->T.
J Cyst Fibros
November 2024
CF Center, Haddasah Medical Center, Jerusalem Israel.
Article Synopsis
- SPL84 is an antisense oligonucleotide (ASO) drug designed to treat cystic fibrosis by correcting splicing issues in the CFTR gene, which are caused by a specific mutation leading to non-functional proteins.
- In a phase 1 clinical trial with 32 healthy individuals, SPL84 was tested for safety and tolerability compared to a placebo, without significant adverse effects observed.
- The results indicated that SPL84 was well-tolerated, with low systemic exposure suggesting it is safe for further development in treating cystic fibrosis.
Promoting readthrough of nonsense mutations in CF mouse model: Biodistribution and efficacy of NV848 in rescuing CFTR protein expression.
Mol Ther
December 2024
Department of Biological, Chemical and Pharmaceutical Sciences and Technologies, University of Palermo, Palermo, Italy. Electronic address:
Article Synopsis
- Nonsense mutations lead to a stop signal in protein production, resulting in non-functional proteins and contributing to diseases like cystic fibrosis (CF).
- This research explores the potential of NV848, a drug designed to promote the continuation of protein synthesis, to enhance CFTR protein expression in a mouse model with a specific CF-causing mutation.
- The study evaluates the drug's stability, distribution in the body, and effectiveness in restoring CFTR protein in mice, suggesting NV848 could be a valuable treatment for CF patients with nonsense mutations.
III BRAZILIAN CONSENSUS STATEMENT ON ENDOSCOPIC ULTRASOUND.
Arq Gastroenterol
October 2024
Universidade de São Paulo, Hospital das Clínicas, Unidade de Endoscopia Gastrointestinal, São Paulo, SP, Brasil.
Background: In the past decades, endoscopic ultrasound has developed from a diagnostic tool to a platform for many therapeutic interventions. Various technological advancements have emerged since the last Brazilian Consensus, demanding a review and update of the recommendations based on the best scientific evidence.
Methods: A group of 32 renowned echoendoscopists selected eight relevant topics to be discussed to generate clinical questions.
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