Problems and solutions in myoblast transfer therapy.
نویسندگان
چکیده
Duchenne muscular dystrophy is a severe X-linked neuromuscular disease that affects approximately 1/3500 live male births in every human population, and is caused by a mutation in the gene that encodes the muscle protein dystrophin. The characterization and cloning of the dystrophin gene in 1987 was a major breakthrough and it was considered that simple replacement of the dystrophin gene would ameliorate the severe and progressive skeletal muscle wasting characteristic of Duchenne muscular dystrophy. After 20 years, attempts at replacing the dystrophin gene either experimentally or clinically have met with little success, but there have been many significant advances in understanding the factors that limit the delivery of a normal dystrophin gene into dystrophic host muscle. This review addresses the host immune response and donor myoblast changes underlying some of the major problems associated with myoblast-mediated dystrophin replacement, presents potential solutions, and outlines other novel therapeutic approaches.
منابع مشابه
The Role of Growth Factors in Myoblast Transfer Therapy
Myoblast transfer therapy, despite unsatisfactory clinical trial results, is still a potential method for the rectification of primary muscle myopathies, provided the procedure can be significantly improved. The technique involves implanting donor myoblasts into diseased muscle by the injection of large numbers of these cells. The majority of transplanted myoblasts die and are rapidly removed f...
متن کاملImmunobiology and the future of myoblast transfer therapy.
Myoblast transfer therapy (MTT) is a cell-mediated gene transfer method aimed at the restoration of normal dystrophin expression in Duchenne muscular dystrophy (DMD). Initial clinical MTT trials were conducted amid much controversy, as they were based on very few animal studies. Unfortunately, the trials were of little therapeutic benefit. As a result, there has been a renaissance of interest i...
متن کاملAbsence of MyoD increases donor myoblast migration into host muscle.
Donor myoblast migration is a major limiting factor in the success of myoblast transfer therapy, a potential treatment for Duchenne muscular dystrophy. A possible strategy to promote the migration of donor myoblasts into host muscle is to enhance their proliferation and delay their fusion, two properties that are major characteristics of myoblasts in regenerating skeletal muscle in MyoD null (-...
متن کاملNon-Fourier heat conduction equation in a sphere; comparison of variational method and inverse Laplace transformation with exact solution
Small scale thermal devices, such as micro heater, have led researchers to consider more accurate models of heat in thermal systems. Moreover, biological applications of heat transfer such as simulation of temperature field in laser surgery is another pathway which urges us to re-examine thermal systems with modern ones. Non-Fourier heat transfer overcomes some shortcomings of Fourier heat tran...
متن کاملNonlinear Analysis of Integrated Kinetics and Heat Transfer Models of Slow Pyrolysis of Biomass Particles using Differential Transformation Method
The inherent nonlinearities in the kinetics and heat transfer models of biomass pyrolysis have led to the applications of various numerical methods in solving the nonlinear problems. However, in order to have physical insights into the phenomena and to show the direct relationships between the parameters of the models, analytical solutions are required. In this work, approximate analytical solu...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
عنوان ژورنال:
- Journal of cellular and molecular medicine
دوره 5 1 شماره
صفحات -
تاریخ انتشار 2001