RNA interference (RNAi) is a sequence-specific gene-silencing mechanism triggered by double-stranded RNA and powerful tools for a gene function study and RNAi therapy. Although siRNAs offer several advantages as potential new drugs to treat various diseases, the efficient delivery system of siRNAs in vivo remains a crucial challenge for achieving the desired RNAi effect in clinical development. In particular, when considering the siRNA therapeutics for familial amyloidotic polyneuropathy (FAP) caused by the deposition of variant transthyretin (TTR) in various organs, hepatocyte-selective siRNA delivery is desired because TTR is predominantly synthesized by hepatocytes. In this study, to reveal the potential use of lactosylated dendrimer (G3)/α-cyclodextrin conjugate (Lac-α-CDE (G3)) as novel hepatocyte-selective siRNA carriers in order to treat FAP, we evaluated the RNAi effect of siRNA complex with Lac-α-CDE (G3) both in vitro and in vivo.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.3109/13506129.2012.674581 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
A Comparative Study of the Electroneurographic Findings in Amyloidotic Polyneuropathy in Patients with Light-Chain Amyloidosis and Glu54Gln Transthyretin Amyloidosis.
Medicina (Kaunas)
December 2024
Fundeni Clinical Institute, 022328 Bucharest, Romania.
: Amyloidosis is a disorder characterized by the abnormal folding of proteins, forming insoluble fibrils that accumulate in tissues and organs. This accumulation disrupts normal tissue architecture and organ function, often with serious consequences, including death if left untreated. Light-chain amyloidosis (AL) and hereditary transthyretin-type amyloidosis (hATTR) are two of the most common types.
View Article and Find Full Text PDFAltered gut microbiota in Taiwanese A97S predominant transthyretin amyloidosis with polyneuropathy.
Sci Rep
March 2024
Departments of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Increasing evidence suggests that gut microbiota alterations are related to development and phenotypes of many neuropsychiatric diseases. Here, we evaluated the fecal microbiota and its clinical correlates in patients with hereditary transthyretin amyloidosis (ATTRv) and polyneuropathy. Fecal microbiota from 38 ATTRv patients and 39 age-matched controls was analyzed by sequencing 16S V3-V4 ribosomal RNA, and its relationships with clinical characteristics of polyneuropathy and cardiomyopathy were explored.
View Article and Find Full Text PDFIatrogenic Amyloid Polyneuropathy Following Domino Liver Transplantation: A Case Report.
Cureus
February 2024
Family Health Unit Cartaxo Terra Viva, Regional Health Administration of Lisbon and Tagus Valley, Cartaxo, PRT.
Familial amyloidotic polyneuropathy (FAP) is an autosomal dominant hereditary disorder. In Portugal, it is mainly linked to transthyretin (TTR) mutation, and patients present with length-dependent sensory-motor polyneuropathy, often accompanied by autonomic dysfunction. Treatment options for FAP include liver transplant, and due to the lack of organs, FAP livers began being implanted in patients with severe liver disease in a process known as domino liver transplantation (DLT).
View Article and Find Full Text PDFClinical outcomes after transplantation of domino grafts or standard deceased donor livers: a systematic review and meta-analysis.
J Gastroenterol Hepatol
April 2024
Transplantation Center, Digestive Disease and Surgery Institute, Department of Immunology, Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio, USA.
Background And Aim: Domino liver transplantation (DLT) utilizes otherwise discarded livers as donor grafts for another recipients. It is unclear whether DLT has less favorable outcomes compared to deceased donor liver transplantation (DDLT). We aimed to assess the outcomes of DLT compared to DDLT.
View Article and Find Full Text PDFDoes Patisiran Reduce Ocular Transthyretin Synthesis? A Pilot Study of Two Cases.
Curr Neuropharmacol
November 2023
Department of Human Neuroscience, Centre for Rare Neuromuscular Disease, Sapienza University of Rome, Rome, Italy.
Background: Variant transthyretin-mediated amyloidosis (ATTR-v) is a well-characterized disease affecting the neurologic and cardiovascular systems. Patisiran has been approved for neurologic involvement as it reduces hepatic synthesis of transthyretin (TTR). Eye involvement is a lateonset feature increasing the risk of glaucoma and cataracts in patients.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!