Gene Therapy - the impossible dream?
Gene Therapy - the impossible dream?

By: stockyard71

See original here:

Gene Therapy - the impossible dream? - Video

One of the main problems with gene therapy is that there is a very low possibility that the plasmid with the new piece of DNA will be correctly inserted into the DNA in the human DNA for several reasons: a) The host cell (bacteria, virus or liposome which has been more recently used) has difficulties in travelling successfully to the human cell in the specific organ or tissue. (e.g.: lungs) If the host cell is a virus, the body can easily destroy them because as the virus touches the surface of the membrane, antibodies will attack it as the cell membrane has glycocalyx which are carbohydrates that can recognize molecules or cells that are not common in the body.

b) It is also because the new piece of DNA has to enter the DNA strand in the correct place. Usually, in a part where the bases are not coding for any protein.

Another negative effect might be that when the new piece of DNA enters the human DNA strand, it can have a terrorific effect on it. This happens when it successfully enters the DNA strand but it replaces or simply disrupts the sequence of amino acids which code for an important protein. (This has occured in the 1980s with the X-SCID disease)

Continued here:

Gene Therapy - Answers.com - Answers - The Most Trusted Place ...

The ProSavin treatment uses an inert virus to carry corrective genes directly into the striatum region of the brain that controls movement.

It is designed to convert ordinary nerve cells into factories for making dopamine, the signalling chemical that is lost in Parkinson's patients.

Loading article content

Lack of dopamine activity leads to the common Parkinson's symptoms of tremor, slow movement and rigidity.

The trial tested the safety, tolerability and effectiveness of three different doses of ProSavin in 15 patients aged 48 to 65 with advanced Parkinson's disease who were not responding to conventional treatments.

A standard system of rating motor functions was used, covering speech, tremors, rigidity, finger taps, posture, gait, and slow movement. Lower scores indicated better muscle control and co-ordination.

Significant score improvements were seen after six months and a year in all patients not taking medication.

Reporting their findings in The Lancet medical journal, the researchers led by Professor Stephane Palfi, from Les Hopitaux Universitaires Henri-Mondor in Creteil, France, wrote: "ProSavin was safe and well tolerated in patients with advanced Parkinson's disease. Improvement in motor behaviour was observed in all patients."

They stressed that, while promising, the results at this stage were still limited and should be "interpreted with caution".

Follow this link:

Gene therapy for Parkinson's produces promising results in first patient trial

10/01/2014 - 07:15:45Back to World Home

A gene therapy for Parkinsons disease has produced promising results in its first patient trial, say researchers.

The ProSavin treatment uses an inert virus to carry corrective genes directly into the striatum region of the brain that controls movement.

It is designed to convert ordinary nerve cells into factories for making dopamine, the signalling chemical that is lost in Parkinsons patients.

Lack of dopamine activity leads to the common Parkinsons symptoms of tremor, slow movement and rigidity.

The trial tested the safety, tolerability and effectiveness of three different doses of ProSavin in 15 patients aged 48 to 65 with advanced Parkinsons disease who were not responding to conventional treatments.

A standard system of rating motor functions was used, covering speech, tremors, rigidity, finger taps, posture, gait, and slow movement. Lower scores indicated better muscle control and co-ordination.

Significant score improvements were seen after six months and a year in all patients not taking medication.

Reporting their findings in The Lancet medical journal, the researchers led by Professor Stephane Palfi, from Les Hopitaux Universitaires Henri-Mondor in Creteil, France, wrote: ProSavin was safe and well tolerated in patients with advanced Parkinsons disease. Improvement in motor behaviour was observed in all patients.

They stressed that, while promising, the results at this stage were still limited and should be interpreted with caution.

Read more from the original source:

Gene therapy provides hope for Parkinson's sufferers

PARIS: A closely-watched prototype therapy to inject corrective genes into the brain to treat Parkinson's disease has cleared an important safety hurdle, doctors said Friday.

Tested on 15 volunteers with an advanced form of the degenerative nerve disease, the technique proved safe and the results were encouraging, they said.

The experiment aims to reverse the lack of a brain chemical called dopamine, which is essential for motor skills.

It entails tucking three genes into a disabled horse virus of the family lentiviruses.

The modified virus is then injected directly into a specialised area of the brain, where it infiltrates cells. In doing so, it delivers corrective pieces of DNA, prompting defective brain cells to once again start producing dopamine.

Called ProSavin, the British-designed treatment was authorised for tests on humans after it was tried on lab monkeys.

It is being closely watched by specialists to see if it works better than conventional therapies -- the veteran drug levodopa or electrical stimulation of the brain -- or another experimental gene technique which uses a modified cold virus.

French neurosurgeon Stephane Palfi, who led the early-stage trial published in The Lancet, said 15 patients aged 48-65 were given the genes in one of three doses.

They developed better coordination and balance, had less muscle twitching and improved speech.

Assessed at least 12 months after the injection, "motor symptoms remained improved in all the patients," Palfi said.

Continued here:

Novel gene therapy for Parkinson's clears hurdle