Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.

نویسندگان

  • Adrian P Kells
  • Piotr Hadaczek
  • Dali Yin
  • John Bringas
  • Vanja Varenika
  • John Forsayeth
  • Krystof S Bankiewicz
چکیده

Transduction of the primate cortex with adeno-associated virus (AAV)-based gene therapy vectors has been challenging, because of the large size of the cortex. We report that a single infusion of AAV2 vector into thalamus results in widespread expression of transgene in the cortex through transduction of widely dispersed thalamocortical projections. This finding has important implications for the treatment of certain genetic and neurodegenerative diseases.

برای دانلود متن کامل این مقاله و بیش از 32 میلیون مقاله دیگر ابتدا ثبت نام کنید

ثبت نام

اگر عضو سایت هستید لطفا وارد حساب کاربری خود شوید

منابع مشابه

Evaluation of Cell Penetrating Peptide Delivery System on HPV16E7 Expression in Three Types of Cell Line

Background: The poor permeability of the plasma and nuclear membranes to DNA plasmids are two major barriers for the development of these therapeutic molecules. Therefore, success in gene therapy approaches depends on the development of efficient and safe non-viral delivery systems. Objectives: The aim of this study was to investigate the in vitro delivery of plasmid DNA encoding HPV16 E7 gene...

متن کامل

نگاهی به ژن درمانی، پیشرفت‏های اخیر و چشم انداز آینده

Human gene therapy has attracted increasing attention as a highly encouraging therapeutic approach to treat wide variety of diseases, other than genetically inherited and monogenic disorders. This approach entails the introduction and expression of a variety of nucleic acids into human target cells for therapeutic purposes. In this article, we review the history, highlights, recently progresses...

متن کامل

Design and bioinformatics analysis of novel biomimetic peptides as nanocarriers for gene transfer

Objective(s): The introduction of nucleic acids into cells for therapeutic objectives is significantly hindered by the size and charge of these molecules and therefore requires efficient vectors that assist cellular uptake. For several years great efforts have been devoted to the study of development of recombinant vectors based on biological domains with potential applications in gene therapy....

متن کامل

The Role of Gene Therapy in Cartilage Repair

The key principle of gene delivery to articulations by direct intra-articular injection is to release complementary DNA(cDNA)-encoding medical products that will lead to maintained, endogenous production of the gene products withinthe articulation. In fact, this has been accomplished for both in vivo and ex vivo gene delivery, using several vectors,genes, and cells in some animal models. Some c...

متن کامل

Short-latency category specific neural responses to human faces in macaque inferotemporal cortex

In this article I would present evidence to show that timing of the flow of neural signals within the ventral visual stream is a crucial part of the neural code for categorization of faces. We recorded the activity of 554 inferotemporal neurons from two macaque monkeys performing a fixation task. More than 1000 object images including human and non-primate animal faces were presented up to 10 t...

متن کامل

ذخیره در منابع من


  با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید

برای دانلود متن کامل این مقاله و بیش از 32 میلیون مقاله دیگر ابتدا ثبت نام کنید

ثبت نام

اگر عضو سایت هستید لطفا وارد حساب کاربری خود شوید

عنوان ژورنال:
  • Proceedings of the National Academy of Sciences of the United States of America

دوره 106 7  شماره 

صفحات  -

تاریخ انتشار 2009