Alpha-1 antitrypsin deficiency: outstanding questions and future directions

M Torres-Durán, JL Lopez-Campos… - Orphanet journal of rare …, 2018 - Springer
Background Alpha-1 antitrypsin deficiency (AATD) is a rare hereditary condition that leads to
decreased circulating alpha-1 antitrypsin (AAT) levels, significantly increasing the risk of …

The lectin–cell interaction and its implications to intestinal lectin-mediated drug delivery

F Gabor, E Bogner, A Weissenboeck, M Wirth - Advanced drug delivery …, 2004 - Elsevier
Based on the fact that oligosaccharides encode biological information, the biorecognition
between lectinised drug delivery systems and glycosylated structures in the intestine can be …

Delivery of mRNA therapeutics for the treatment of hepatic diseases

Z Trepotec, E Lichtenegger, C Plank, MK Aneja… - Molecular Therapy, 2019 - cell.com
Promising improvements in the field of transcript therapeutics have clearly enhanced the
potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are …

RNAi for treating hepatitis B viral infection

Y Chen, G Cheng, RI Mahato - Pharmaceutical research, 2008 - Springer
Chronic hepatitis B virus (HBV) infection is one of the leading causes of liver cirrhosis and
hepatocellular carcinoma (HCC). Current treatment strategies of HBV infection including the …

[图书][B] Animal lectins: form, function and clinical applications

GS Gupta - 2012 - books.google.com
Animal Lectins: Form, Function and Clinical Applications presents up-to-date knowledge of
animal lectins. Detailed descriptions on biological activities, tissue and/or subcellular …

Genetic modification of the lung directed toward treatment of human disease

D Sondhi, KM Stiles, BP De, RG Crystal - Human gene therapy, 2017 - liebertpub.com
Genetic modification therapy is a promising therapeutic strategy for many diseases of the
lung intractable to other treatments. Lung gene therapy has been the subject of numerous …

[图书][B] Handbook of clinical nanomedicine: nanoparticles, imaging, therapy, and clinical applications

R Bawa, GF Audette, I Rubinstein - 2016 - books.google.com
This handbook (55 chapters) provides a comprehensive roadmap of basic research in
nanomedicine as well as clinical applications. However, unlike other texts in nanomedicine …

Gene therapy for alpha-1 antitrypsin deficiency lung disease

MJ Chiuchiolo, RG Crystal - Annals of the American Thoracic …, 2016 - atsjournals.org
Alpha-1 antitrypsin (AAT) deficiency, characterized by low plasma levels of the serine
protease inhibitor AAT, is associated with emphysema secondary to insufficient protection of …

Hydrodynamic liver gene transfer mechanism involves transient sinusoidal blood stasis and massive hepatocyte endocytic vesicles

A Crespo, A Peydro, F Dasi, M Benet, JJ Calvete… - Gene Therapy, 2005 - nature.com
The present study contributes to clarify the mechanism underlying the high efficacy of
hepatocyte gene transfer mediated by hydrodynamic injection. Gene transfer experiments …

[图书][B] Polymeric gene delivery: principles and applications

MM Amiji - 2004 - books.google.com
To treat disease or correct genetic disorders using gene therapy, the most suitable vehicle
must be able to deliver genes to the appropriate tissues and cells in the body in a specific as …