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Review of Genetic and Gene Therapy for Parkinson’s Disease
vFollowing Alzheimer's, PD has the highest number of neuronal degeneration among all neurodegenerative disorders. Deficiency of dopamine in the basal ganglia precedents motor symptoms, viz, later postural unsteadiness, rigidity, tremor, and bradykinesia. PD in most cases is instigated by an amalgamation
vFollowing Alzheimer's, PD has the highest number of neuronal degeneration among all neurodegenerative disorders. Deficiency of dopamine in the basal ganglia precedents motor symptoms, viz, later postural unsteadiness, rigidity, tremor, and bradykinesia. PD in most cases is instigated by an amalgamation
Farooqji
in
Cure Parkinson's
2 years ago
NURR1: Facilitating the transition of neural stem cells into functional dopaminergic neurons.
//////What is NURR1? Nuclear receptor related 1 protein (Nurr1) is an important transcription factor required for differentiation and maintenance of midbrain dopaminergic (DA) neurons. Since decrease in Nurr1 function either due to diminished expression or rare mutation is associated with Parkinson's
//////What is NURR1? Nuclear receptor related 1 protein (Nurr1) is an important transcription factor required for differentiation and maintenance of midbrain dopaminergic (DA) neurons. Since decrease in Nurr1 function either due to diminished expression or rare mutation is associated with Parkinson's
House2
in
Cure Parkinson's
2 years ago
New gene therapy delivers treatment directly to brain
https://www.pbs.org/newshour/science/new-gene-therapy-delivers-treatment-directly-to-brain
https://www.pbs.org/newshour/science/new-gene-therapy-delivers-treatment-directly-to-brain
Farooqji
in
Cure Parkinson's
2 years ago
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jakafi, PV and retinal tears and detachment
in May I had a small stroke and the same day a retinal detachment. I was released from hospital 3 days later directly to an eye surgeon to repair my eye. I developed rapidly growing cataracts as a resulting complication. Last month I had surgery to remove cataracts in both eyes and now am having almost
in May I had a small stroke and the same day a retinal detachment. I was released from hospital 3 days later directly to an eye surgeon to repair my eye. I developed rapidly growing cataracts as a resulting complication. Last month I had surgery to remove cataracts in both eyes and now am having almost
DDSmiley63
in
Fight MPN
2 years ago
Getting ready to try a neurogenesis protocol.
I think I've got the neuroprotection and neurorehab down pretty well. Next up, neurogenesis... Until recently, neuroscientists believed that the central nervous system, including the brain, was incapable of neurogenesis and unable to regenerate. However, stem cells were discovered in parts of the adult
I think I've got the neuroprotection and neurorehab down pretty well. Next up, neurogenesis... Until recently, neuroscientists believed that the central nervous system, including the brain, was incapable of neurogenesis and unable to regenerate. However, stem cells were discovered in parts of the adult
House2
in
Cure Parkinson's
2 years ago
Revolutionary gene therapy offers hope for untreatable cancers
This is pretty fundamental Seems like there should be no reason this would not work with prostate cancer Revolutionary gene therapy offers hope for untreatable cancers | Cancer research | The Guardian Https://www.theguardian.com/science/2022/dec/11/revolutionary-gene-therapy-offers-hope-untreatable-cancers
This is pretty fundamental Seems like there should be no reason this would not work with prostate cancer Revolutionary gene therapy offers hope for untreatable cancers | Cancer research | The Guardian Https://www.theguardian.com/science/2022/dec/11/revolutionary-gene-therapy-offers-hope-untreatable-cancers
cesces
in
Advanced Prostate Cancer
2 years ago
How does regret affect your ability to cope with stress? - study
[i]
“We discovered that stress-susceptible mice were hypersensitive to type- one regret and insensitive to type-two regret'' (credit: Dr. Brian Sweis)
[/i] The brain’s sensitivity to different types of regret may impact mood disorders like depression. By JUDY SIEGEL-ITZKOVICH Published: OCTOBER
[i]
“We discovered that stress-susceptible mice were hypersensitive to type- one regret and insensitive to type-two regret'' (credit: Dr. Brian Sweis)
[/i] The brain’s sensitivity to different types of regret may impact mood disorders like depression. By JUDY SIEGEL-ITZKOVICH Published: OCTOBER
lupus-support1
Administrator
in
LUpus Patients Understanding and Support
2 years ago
Upstaza became the first approved therapy to treat a rare genetic condition. It would seem that this should be encouraging for Parkinson's
"In July this year, Upstaza became the first approved disease-modifying treatment for AADC deficiency and the first marketed gene therapy directly infused into the brain when it was authorized by the European Medicines Agency." In Parkinson's cases where there is a genetic problem, this is encouraging
"In July this year, Upstaza became the first approved disease-modifying treatment for AADC deficiency and the first marketed gene therapy directly infused into the brain when it was authorized by the European Medicines Agency." In Parkinson's cases where there is a genetic problem, this is encouraging
Zardoz
in
Cure Parkinson's
2 years ago
Rapidly rising PSA and new bone met(s).
Complete case history in the profile.
07/24/22 PSA - 0.97 08/24/22 PSA - 1.31 09/15/22 - PSA - 1.69 - Total Testosterone 23.38 ng/dl ( edit: mistyped as free earlier) Currently on 3 month lupron (since 08/22 ) and abirateron ( since 10/21 ) + radiation. New PSMA has shown a (first)
Complete case history in the profile.
07/24/22 PSA - 0.97 08/24/22 PSA - 1.31 09/15/22 - PSA - 1.69 - Total Testosterone 23.38 ng/dl ( edit: mistyped as free earlier) Currently on 3 month lupron (since 08/22 ) and abirateron ( since 10/21 ) + radiation. New PSMA has shown a (first)
meowlicious99
in
Advanced Prostate Cancer
2 years ago
Million-Dollar Drug:
The FDA approved a gene therapy from Bluebird Bio that represents a potential cure for a rare blood disorder—at a price of $2.8 million per patient. Will anyone be able to afford the cure if it comes down the pipe from this source?
The FDA approved a gene therapy from Bluebird Bio that represents a potential cure for a rare blood disorder—at a price of $2.8 million per patient. Will anyone be able to afford the cure if it comes down the pipe from this source?
Scout4answers
in
Advanced Prostate Cancer
2 years ago
A Gene that affects allele reduction in Jak2 by IFN.
I came across this article. It's an example of experts continuing to slice and dice the Ropeg studies. https://ashpublications.org/blood/article/137/3/387/463332/Germline-genetic-factors-influence-the-outcome-of It looks like a careful study and meets my quality test req't that I can follow it well
I came across this article. It's an example of experts continuing to slice and dice the Ropeg studies. https://ashpublications.org/blood/article/137/3/387/463332/Germline-genetic-factors-influence-the-outcome-of It looks like a careful study and meets my quality test req't that I can follow it well
EPguy
in
MPN Voice
2 years ago
Hesitant to start Endep
I have severe rls and suffer augmentation. I am trying to slowly wean off sifrol and have been taking 400mg gabapentan.I take 30mg codeine in early hours of each morning(1-3am)to ease rls and help me go back tap sleep.I also have fibromyalgia.My sleep specialist wants me to start endep to help my sleep
I have severe rls and suffer augmentation. I am trying to slowly wean off sifrol and have been taking 400mg gabapentan.I take 30mg codeine in early hours of each morning(1-3am)to ease rls and help me go back tap sleep.I also have fibromyalgia.My sleep specialist wants me to start endep to help my sleep
Retiredlady
in
Restless Legs Syndrome
2 years ago
Swanbio Gene Therapy Trial
Has anyone researched the swanbio gene therapy trial? Or even began the trial? I was wondering how often you would have to go to Boston etc. I can get around but my balance isn’t great and am getting weaker. Wondering if it would basically halt the progression or possibly make it a little better? I
Has anyone researched the swanbio gene therapy trial? Or even began the trial? I was wondering how often you would have to go to Boston etc. I can get around but my balance isn’t great and am getting weaker. Wondering if it would basically halt the progression or possibly make it a little better? I
Golfdog3
in
AMN EASIER
2 years ago
Watch Prof Tom Foltynie on the topic of repurposing diabetes drugs for Parkinson's disease - Recording now available
Thank you to Prof Tom Foltynie for his time and expertise last night on the topic of repurposing diabetes drugs, including Exenatide, for Parkinson's disease. It was an excellent presentation and discussion that brings us all up to date with the latest in the trials, including feedback from some of you
Thank you to Prof Tom Foltynie for his time and expertise last night on the topic of repurposing diabetes drugs, including Exenatide, for Parkinson's disease. It was an excellent presentation and discussion that brings us all up to date with the latest in the trials, including feedback from some of you
Michel0220
in
Cure Parkinson's
2 years ago
Seelos Therapeutics announces statistically significant downregulation of mRNA & ~40% reduction of PD alpha synuclein
Seelos Therapeutics Announces Data Demonstrating Statistically Significant Downregulation of mRNA and Reduction of Alpha Synuclein in an In Vitro Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 in Dementia with Lewy Bodies
Preliminary Findings of In Vitro Study
The goal of this in vitro study
Seelos Therapeutics Announces Data Demonstrating Statistically Significant Downregulation of mRNA and Reduction of Alpha Synuclein in an In Vitro Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 in Dementia with Lewy Bodies
Preliminary Findings of In Vitro Study
The goal of this in vitro study
Hidden
in
Cure Parkinson's
2 years ago
Prof Tom Foltynie: “Does the repurposing of diabetes drugs offer hope for Parkinson’s patients?” - Wed 29th June at 7.30 pm London time
I am delighted to announce our next zoom session that will be taking place at 7.30 pm London time on Wednesday, the 29th of June. We will have the pleasure of listening to Professor Tom Foltynie who will talk to us about “Does the repurposing of diabetes drugs offer hope for Parkinson’s patients?”
I am delighted to announce our next zoom session that will be taking place at 7.30 pm London time on Wednesday, the 29th of June. We will have the pleasure of listening to Professor Tom Foltynie who will talk to us about “Does the repurposing of diabetes drugs offer hope for Parkinson’s patients?”
Michel0220
in
Cure Parkinson's
2 years ago
Gene therapy study starts for bone marrow failure in dyskeratosis congenita (DC)
Dr Kasiani Myers, from Cincinnati has treated the first patient with DC-related bone marrow failure, with an experimental new gene therapy (EXG34217) The trial is in its very early, safety testing stages but we hope that the treatment will help treat bone marrow failure (low blood counts) The trial is
Dr Kasiani Myers, from Cincinnati has treated the first patient with DC-related bone marrow failure, with an experimental new gene therapy (EXG34217) The trial is in its very early, safety testing stages but we hope that the treatment will help treat bone marrow failure (low blood counts) The trial is
JayKayPee
Administrator
in
DC Action
2 years ago
Metachromatic leukodystrophy and the world's most expensive drug.
I'll post this. It's tangentially related to our disease. From The Guardian:
[u]Children in England with fatal condition to get world’s most expensive drug.[/u]
https://www.theguardian.com/society/2022/feb/04/children-in-england-with-fatal-condition-to-get-worlds-most-expensive-drug "Children
I'll post this. It's tangentially related to our disease. From The Guardian:
[u]Children in England with fatal condition to get world’s most expensive drug.[/u]
https://www.theguardian.com/society/2022/feb/04/children-in-england-with-fatal-condition-to-get-worlds-most-expensive-drug "Children
monkeybus
in
AMN EASIER
3 years ago
CLL patient cured a decade after gene therapy
In 2010, doctors treated Doug Olson’s leukemia with an experimental gene therapy that transformed some of his blood cells into cancer killers. More than a decade later, there’s no sign of cancer in his body. The treatment cured Olson and a second patient, according to the University of Pennsylvania doctors
In 2010, doctors treated Doug Olson’s leukemia with an experimental gene therapy that transformed some of his blood cells into cancer killers. More than a decade later, there’s no sign of cancer in his body. The treatment cured Olson and a second patient, according to the University of Pennsylvania doctors
WinJ3
in
CLL Support
3 years ago
Gene therapy for genetic disorder affecting brain dopamine metabolism shows success; same delivery technique being investigated for PD
Really exciting report of disease reversal in children with AADC (aromatic L-amino acid decarboxylase deficiency); article mentions same delivery technique is being used for GDNF gene therapy in Parkinson's. https://wexnermedical.osu.edu/departments/innovations/neuronews/gene-therapy-new-approach
Really exciting report of disease reversal in children with AADC (aromatic L-amino acid decarboxylase deficiency); article mentions same delivery technique is being used for GDNF gene therapy in Parkinson's. https://wexnermedical.osu.edu/departments/innovations/neuronews/gene-therapy-new-approach
Rhyothemis
in
Cure Parkinson's
3 years ago
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