Gene Therapies For Pd And Hd
For the development of new therapies for PD and HD, it is important to include, especially for HD and genetic forms of PD, genetic correction/editing of the mutated gene. Nowadays, there are several gene silencing/editing technologies, including RNA interference , antisense oligonucleotides , and clustered interspaced short palindromic repeats , which can be used as therapies for the treatment of PD and HD. For a more in-depth knowledge of gene therapy delivery systems and other cellular targets, reviews are published elsewhere .
As previously stated, PD is characterized by the selective degeneration of dopaminergic neurons in the SN, thus approaches aiming to revert this loss based on the delivery of genes encoding for enzymes required for DA synthesis could be useful. The first enzyme for DA synthesis is TH, which requires the enzyme GTP-cyclohydrolase-1 to synthesize a cofactor for DA biosynthesis . TH converts tyrosine into L-dopa, which finally is converted into DA by the aromatic L-amino acid decarboxylase . Therapies to deliver enzymes involved in DA synthesis have been proved in preclinical and clinical studies showing its benefits.
When Should I Call My Healthcare Provider
If you have Huntington disease, it is important to closely monitor you condition, and contact your healthcare provider if you notice any of the following symptoms:
- Increasing difficulty with motor tasks such as walking
- Trouble swallowing
- Mood changes, such as increasing symptoms of depression or feelings of suicide
What Are The Risk Factors For Huntington Disease
If you have the Huntington disease gene, you will develop the disease at some point during your life. The age of onset of Huntington disease varies greatly from person to person, but most people develop it in their 30s or 40s.
Huntington disease is a rare disorder. More than 15,000 Americans currently have the disease, but many more are at risk of developing it.
Huntingtons Disease | Generations of Care and Search for a Cure
The Johns Hopkins Huntingtons Disease Center for Excellence is honored to have served multiple generation of families for the past forty years in HD history. Join neurologist Jee Bang, Clinical Director of Johns Hopkins Huntington Disease Center of Excellence, to learn more about Huntingtons Disease and efforts underway for families affected by the disease now and in the future.
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Key Difference Parkinsons Vs Huntingtons Disease
The key difference between Parkinsons and Huntingtons disease is that Parkinson disease is a disorder with rigidity, tremors, slowing of movements, postural instability and gait disturbances usually occurring in old age due to degeneration of the substantia nigra of the midbrain while Huntingtons disease is a familial neurodegenerative disorder usually occurring in a younger population, characterized by emotional problems, loss of thinking ability and abnormal choreiform movements .
Overview Of Parkinsons Disease
According to the American Academy of Physical Medicine and Rehabilitation, Parkinsons Disease is a progressive movement disorder resulting from the loss of nerve cells in the brain that produce a substance called dopamine. The cause of Parkinsons Disease is unknown. One in every 800 individuals develops the condition, which is more common in men than in women. Parkinsons Disease increases with age, with 90% of patients above 45 years of age, and may be related to genetic mutations. Exercise throughout adulthood may reduce a persons risk.
Early symptoms of Parkinsons Disease begin on one side of the body, with diminished fine motor control, or reduced foot or arm movement. Other symptoms include tremors, stiffness, posture imbalance, difficulty swallowing, constipation and urinary incontinence. Patients may experience slower movement and speech, fatigue, forgetfulness, loss of smell, and sleep disorders. Symptoms of late-stage Parkinsons Disease include an expressionless face, soft voice and stooped posture, and an inability to walk.
Healthcare providers conduct thorough physical exams, noting specific signs and symptoms that are seen in Parkinsons Disease. Diagnostic testing may play a role in evaluating the disease. Sleep studies, swallowing studies and psychological testing also can assess the severity of the condition.
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Essential Tremor Is Different From Parkinsons Disease And Often Far Less Serious
Benign Essential Tremor vs. Parkinsons Disease: Whats the Difference?
No one wishes for a chronic, long-lasting medical condition but some are more dreaded than others especially if theres no cure. Diseases that involve neurological deterioration are particularly fearful. They affect the brain and/or nervous system. They include deteriorating conditions like MS, ALS, Huntingtons disease, Alzheimers disease, Parkinsons disease and others.
When a person develops an involuntary rhythmic shaking it is normal to be worried because a tremor of the hands, head or other part of the body can be a symptom of Parkinsons disease . However, the most common involuntary movement disorder, benign essential tremor , is in a neurological class of its own. It is estimated that 4% of people over 40 have ET, affecting at least 10 million North Americans. Unlike PD, it is more apt to cause minor impairments, inconvenience, and embarrassment.
Nonetheless, when trembling of the hands, face or neck begins, it is common for patients to worry that they have PD. There are several ways to tell PD from ET, and specialists called neurologists are trained to tell which is which, thus putting the minds of ET patients at rest.
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Causes Of Huntington’s Disease
In 1993, researchers found the gene that causes Huntington’s. Everyone has the HD gene, but in some families an abnormal copy of the gene gets passed from parent to child. If you have a parent with Huntington’s disease, you have a 50% chance of having the gene and developing the disease.
Men and women are equally likely to inherit the abnormal gene.
If you don’t have the abnormal gene, you can’t get Huntington’s or pass it on to your children.
The disease doesn’t skip generations.
If you or members of your family plan to be tested for Huntington’s, it’s a good idea to get professional genetic counseling first. Counselors can help explain what to expect from the test results.
With knowledge about the HD gene, scientists have been able to learn a great deal about how the disease affects the brain. More importantly, this discovery may help pave the way for future treatment.
Huntington’s Disease Society of America: “Huntington’s Disease: A Family Guide,” “Stages of HD,” and “Nutrition and HD: Huntington’s Disease.”
National Institute of Neurological Disorders and Stroke: “NINDS Huntington’s Disease Information Page.”
Alzheimer’s Association: “Huntington’s Disease.”
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Comparison Table Between Huntingtons And Parkinsons
|Parameters of Comparison|
or magnetic resonance imaging Magnetic Resonance Imaging In magnetic resonance imaging , a strong magnetic field and very high frequency radio waves are used to produce highly detailed images. MRI does not use x-rays and is usually very safe read more is done to check for the degeneration of the basal ganglia and other areas of the brain usually affected by the disease and to rule out other disorders.
Genetic testing is done to confirm the diagnosis. Genetic testing and counseling are important for people who have a family history of the disease but no symptoms because people are likely to have children before symptoms appear. For such people, genetic counseling should precede genetic testing. They are referred to centers that have expertise in dealing with the complex ethical and psychologic issues involved.
How Are They Alike
These diseases both affect your nerves. MS can break down the coating, called myelin, that surrounds and protects your nerves. In Parkinsonâs, nerve cells in a part of your brain slowly die off.
Both can start out with mild symptoms, but they get worse over time.
Common symptoms of both diseases include:
- Shaky fingers, hands, lips, or limbs
- Slurred speech thatâs hard for others to understand
- Numb or weak limbs that make your walk unsteady
- Loss of muscle control that often affects one side of your body at first, then later both
- Spastic limb movements that are hard to control
- Loss of bladder or bowel control
Depression is another symptom common to both conditions.
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Symptoms Of Huntington’s Disease
Although symptoms may first show up in midlife, Huntington’s can strike anyone from childhood to advanced age. Symptoms often first appear when people are in their 30s or 40s. Over 10 to 25 years, the disease gradually kills nerve cells in the brain. This affects the body, mind, and emotions. If symptoms begin before age 20, itâs called juvenile Huntingtonâs Disease and it may get worse faster.
Symptoms can vary greatly from person to person. And stress or excitement can worsen symptoms.
Some symptoms are easier to spot than others. Abnormal movements may be the first thing you notice. Weight loss can be a concern at all stages.
Symptoms of Huntington’s disease tend to develop in stages.
Early stage symptoms
Changes may be quite subtle in early stages, making it possible to keep driving and working. You may just require a little extra help.
Common early symptoms include:
How Is Huntington Disease Treated
You cant cure or slow the progression of Huntington disease, but health care providers can offer medications to help with certain symptoms.
Drugs like haloperidol, tetrabenazine, and amantadine are especially helpful for controlling the unusual movements caused by Huntington disease. Haloperidol and tetrabenazine can also help offset hallucinations and delusional thoughts. Depression and suicide are common among those with Huntington disease. Antidepressants and antianxiety medications may be prescribed to treat these symptoms.
On The Right Track To Treat Movement Disorders: Promising Therapeutic Approaches For Parkinsons And Huntingtons Disease
- 1Center for Integrative Biology, Faculty of Sciences, Universidad Mayor, Santiago, Chile
- 2Faculty of Medicine, Biomedical Neuroscience Institute, University of Chile, Santiago, Chile
- 3Program of Cellular and Molecular Biology, Institute of Biomedical Sciences, University of Chile, Santiago, Chile
- 4Center for Geroscience, Brain Health, and Metabolism, University of Chile, Santiago, Chile
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Is Parkinsons Disease Treated By A Neurologist
People with Parkinsons disease will usually require a team of healthcare professionals to help them manage the condition.
A neurologist, a doctor specializing in conditions of the brain and nervous system, will be one of the main people involved in treating Parkinsons.
Other healthcare professionals who may help treat Parkinsons can include:
- a persons regular doctor
- a physical therapist
- a speech or occupational therapist
- mental health professional
- other specialists, such as a gastroenterologist, if people experience other symptoms of Parkinsons such as digestive issues
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Patient Groups Versus Controls
There were significant relative differences in vibration-evoked rCBF between patients and controls that can be summarized as follows: in Huntington’s disease, relatively decreased activation of contralateral S2 , S1 , parietal areas 39 and 40 , and lingual gyrus , bilateral prefrontal cortical areas 8, 9, 10 and 44 , along with decreased activation of contralateral basal ganglia in Parkinson’s disease, relatively decreased activation of contralateral sensorimotor cortex , lateral premotor cortex , S2 , contralateral posterior cingulate , bilateral prefrontal cortex and contralateral basal ganglia in both entities, relatively enhanced activation of ipsilateral sensory cortical areas, notably caudal S1 , S2 and insular cortex . The data are summarized in Table 6 and Table 7 .
Human Fetal Tissue As A Source Of Progenitor Cells
The first study demonstrating that dopaminergic neurons could be replaced using fetal tissue was performed using 6-hydroxydopamine -lesioned rats that were implanted with DA-rich ventral mesencephalic tissue from rat fetuses . These studies were followed by the generation of the first non-human primates PD model: monkeys lesioned with 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine . This model manifested several of the patients symptoms, and transplanting primate fetal mesencephalic tissue into their striatum showed to alleviate these symptoms . These studies set foot for the first PD cell replacement therapy in humans. These clinical trials were performed using dopaminergic neuron precursors from human fetal tissue, which were transplanted into the striatum of PD patients . Transplanted tissue presented no negative effects at the transplantation site, was functional and survived in the transplanted brain region, but clinical benefits were variable .
Table 3. Common animal models of Parkinsons Disease.
Table 4. Common animal models of Huntingtons disease.
Cellular Replacement Therapies For Pd And Hd
In 1967, in an important breakthrough, Cotzias et al. demonstrated that the administration of a precursor of DA, L-dopa, improved motor function in PD patients, leading to the thought that the cure for PD was discovered. Also in the 1960s, tetrabenazine was introduced as an antipsychotic but also showed beneficial effects for the treatment of hyperkinetic motor symptoms, like chorea in HD patients . To date, it is known that these drugs do not reverse disease progression and in many cases do not have the desired effects. This has brought the idea that local production of DA and GABA, and therefore the replacement of the neurons that produce it, would be the ideal treatment for these diseases. The fact that the major symptoms present in PD and HD patients are due to the loss of dopaminergic and GABAergic neurons in specific brain regions, respectively, means that replacing these specific cell types could help relieve some of the symptoms present in patients. This has given rise to different branches of investigations seeking cellular replacement-based therapies, which have shown promising results in animal models for these diseases as well as in affected patients .
Cellular Reprogramming For Pd And Hd
In the adult brain, NSCs are present in the subventricular zone of the lateral ventricle and the subgranular zone of the dentate gyrus. These NSCs are capable of generating neuroblasts, which differentiate into mature neurons . Despite the presence of a niche for the generation of new neurons, these cells have limited migration to remote regions, like the SN and striatum. Hence, the idea to generate new local neurons from preexisting cells has been studied for the last 10 years. Initial studies have demonstrated that fibroblasts can be reprogrammed to dopaminergic neurons through the ectopic expression of transcription factors . Considering the reprogramming of cells for the treatment of neurodegenerative diseases, astrocytes were initially considered as an attractive alternative and their reprogramming to neurons forming functional synapses was demonstrated .
Recently, using AAV-based reprogramming of striatal astrocytes, Wu et al. demonstrated that astrocytes could be converted to MSNs in the striatum of R6/2 and YAC128 mice. Converted neurons expressed specific MSNs markers, showed electrophysiological properties, and projected their axonal terminals to the GP and SNpr. All these findings were accompanied by a reduction in striatal atrophy, attenuation of the phenotypic deficit, and an extended life span of R6/2 mice with converted MSNs .
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Huntingtons Disease Dementiaa Not
If and when the day comes that I may forget who you are, please promise that you wont forget that I am still me.
Remember the song This Land Is Your Land? It was written by Woody Guthrie.
In the 1940s, his behavior changed violently, affecting his work, his relationships, his life. It wasnt till the early 1950s that they figured out why.
He had Huntingtons Disease Dementia.
When Do Huntingtons Disease Dementia Symptoms Start?
Huntingtons Disease is much rarer than many of the other dementias weve talked about. In 2019, statistics stated HD diagnoses as
- 2.7 people per 100,000 worldwide,
- 10 per 100,000 in Europe,
- 9 per 100,000 in America, and
- fascinatingly, 21 per 100,000 in Egypt.
For those who have it, symptoms usually show up in their 30s and 40s, but they can start younger, even as early as two years old, or as late as 80.
What Are the Symptoms of Huntingtons Disease Dementia?
Like Parkinsons, Huntingtons Disease is first a motor disease.
The biggest recognizable symptom?
- Involuntary, uncontrolled, jerky, flailed movements of the arms, legs, head, face, and upper body.
But it also weakens
All Huntingtons symptoms start out mild in its early stage. The person can generally care for his own needs . But by the middle stage, he may need help.
What Causes Huntingtons Disease Dementia?
Different from what we know about Parkinsons, Alzheimers, Lewy Body, and vascular dementias, Huntingtons Disease is a genetic disorder.
No more messages, no more calm.
Parkinsons Disease Vs Als: Us Prevalence
One million Americans live with Parkinsons disease. The average cost of Parkinsons disease including treatment, lost work wages, and social security payments is $25 billion annually in the U.S.
It is not clear how many people are affected by ALS, but the estimates range between 12,000 and 15,000. Doctors tell roughly 5,000 patients annually that they have ALS. Records on ALS have not been well kept across the country, so estimates may fall way below the actual rates. Common age of ALS diagnosis is between 55 and 75, and life expectancy is anywhere between two and five years after the onset of symptoms. Longevity in ALS is strongly linked to a persons age. Younger individuals with ALS tend to live longer than those diagnosed at an older age.
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Key Difference Huntingtons Disease Vs Alzheimers
Huntingtons disease is a cause of chorea, usually appearing in the middle years of life and later becoming complicated with psychiatric and cognitive abnormalities. Alzheimers disease is a neurodegenerative condition of the brain, which is characterized by the atrophy of brain tissues and it has been identified as the most common cause of dementia. In Huntingtons disease, there is a predominant motor impairment which is not observed in the Alzheimers disease. This is the key difference between Huntingtons disease and Alzheimers.