01-09-2014, 06:32 AM
Lysosomal storage diseases known as LSDs is a series of genetic disorders owing to problems in any aspect of lysosomal biology. In the last two decades different treatments have been introduced to deal with these conditions. And, the most famous among them is enzyme replacement therapy ERT. Yes some of these disorders have been successfully treated with enzyme replacement therapy.
But, it must be understood that this therapy has some limitations like biodistribution of recombinant enzymes and too much financial cost.
Recently a new strategy for the treatment of LSDs has been launched and it is called as pharmacological chaperone therapy PCT. It is based on chaperone molecules which helps the folding of mutated enzymes. Yes, their functionality and lysosomal trafficking are improved through this strategy.
After in-depth studies, pharmacological chaperone therapy is supposed to be translated into clinical applications for dealing with Gaucher, Pompe , and Fabry diseases.
This strategy could only be used on those patients having chaperone responsive mutations.
Recently, a demonstration has been carried to asses whether these kinds of therapies have any negative impacts or not. It has also been determined that whether PCT is worth using in clinical applications.
This demonstration moots the drawbacks and strengths of the above mentioned therapies available for Lysosomal storage diseases and suggests that a research in future should be directed towards the improvement of treatment protocols. Besides, it has also been proposed that combination of different available therapies should also be investigated for determining the outsome of Lsds patients.
It goes without saying that this enzyme replacement therapy had been suggested by De Dude in the early years of 1960s. He said that these Lsds diseases could be diagnosed with the advent of genetic molecular technique. On the contrary, in the early years of 1980s, Hobbs proposed that Bone marrow transplantation could also be used for combating against Lysosomal storage diseases.
But, it must be understood that this therapy has some limitations like biodistribution of recombinant enzymes and too much financial cost.
Recently a new strategy for the treatment of LSDs has been launched and it is called as pharmacological chaperone therapy PCT. It is based on chaperone molecules which helps the folding of mutated enzymes. Yes, their functionality and lysosomal trafficking are improved through this strategy.
After in-depth studies, pharmacological chaperone therapy is supposed to be translated into clinical applications for dealing with Gaucher, Pompe , and Fabry diseases.
This strategy could only be used on those patients having chaperone responsive mutations.
Recently, a demonstration has been carried to asses whether these kinds of therapies have any negative impacts or not. It has also been determined that whether PCT is worth using in clinical applications.
This demonstration moots the drawbacks and strengths of the above mentioned therapies available for Lysosomal storage diseases and suggests that a research in future should be directed towards the improvement of treatment protocols. Besides, it has also been proposed that combination of different available therapies should also be investigated for determining the outsome of Lsds patients.
It goes without saying that this enzyme replacement therapy had been suggested by De Dude in the early years of 1960s. He said that these Lsds diseases could be diagnosed with the advent of genetic molecular technique. On the contrary, in the early years of 1980s, Hobbs proposed that Bone marrow transplantation could also be used for combating against Lysosomal storage diseases.
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