Gasser, T. (2001). "Genetics of Parkinson's disease." J
Neurol248(10): 833-40.
Over the past few years, several genes for monogenically inherited forms of
Parkinson's disease (PD) have been mapped and/or cloned. In a small number of
families with autosomal dominant inheritance and typical Lewy-body pathology,
mutations have been identified in the gene for alpha-synuclein. Aggregation of
this protein in Lewy-bodies may be a crucial step in the molecular pathogenesis
of familial and sporadic PD. On the other hand, mutations in the parkin gene
cause autosomal recessive parkinsonism of early onset. In this form of PD,
nigral degeneration is not accompanied by Lewy-body formation. Parkin-mutations
appear to be a common cause of PD in patients with very early onset. Parkin has
been implicated in the cellular protein degradation pathways, as it has been
shown that it functions as a ubiquitin ligase. The potential importance of this
pathway is also highlighted by the finding of a mutation in the gene for
ubiquitin C-terminal hydrolase L1 in another small family with PD. Other loci
have been mapped to chromosome 2p and 4p, respectively, in a small number of
families with dominantly inherited PD, but those genes have not yet been
identified. These findings prove that there are several genetically distinct
forms of PD that can be caused by mutations in single genes. On the other hand,
there is at present no direct evidence that any of these genes have a direct
role in the aetiology of the common sporadic form of PD. Epidemiological, case
control, and twin studies, although supporting a genetic contribution to the
development of PD, all suggest a clear familial clustering only in a minority of
cases. It is therefore widely believed that a combination of interacting genetic
and environmental causes may be responsible in this majority of PD-cases.
However, studies of gene-environment interactions have not yet produced any
convincing results. Nevertheless, the elucidation of the molecular sequence of
events leading to nigral degeneration in clearly inherited cases is likely to
shed light also on the molecular pathogenesis of the common sporadic form of
this disorder.
Klein, C. (2001). "[The genetics of Parkinson syndrome]." Schweiz Rundsch Med
Prax90(23): 1015-23.
A genetic contribution to the etiology of Parkinson's disease was first
suspected by Charcot and later confirmed by case control, family, and twin
studies, as well as by the description of large parkinsonian families with
Mendelian inheritance of the disease. Recent progress in the field of molecular
neurogenetics has led to the identification of several Parkinson disease genes
and gene loci. Mutations in the alpha-Synuclein gene (PARK1) and in the gene for
the ubiquitin C-terminal hydrolase I (PARK5), along with two gene loci harboring
currently unknown genes (PARK3 and PARK4), have been linked to very rare
autosomal dominantly inherited parkinsonian syndromes. Mutations in the parkins
gene (PARK2), causing autosomal recessive early-onset parkinsonism, are much
more common and therefore of clinical relevance. A second gene locus for an
autosomal dominantly inherited Parkinsonian syndrome was recently localized on
chromosome 1 (PARK6). All three parkinson genes identified thus far imply the
involvement of the ubiquitin pathway of protein degradation in the pathogenesis
of Parkinson's disease.
Lev, N. and E. Melamed (2001). "Heredity in Parkinson's disease: new findings."
Isr Med Assoc J3(6): 435-8.
Multiple factors have been hypothesized over the last century to be causative or
contributory for Parkinson's disease. Hereditary factors have recently emerged
as a major focus of Parkinson's disease research. Until recently most of the
research on the etiology of Parkinson's disease concentrated on environmental
factors, and the possibility that genetic factors contribute significantly to
the pathogenesis of Parkinson's disease has been neglected. However, it has
become increasingly apparent that even in sporadic cases, the disease most
likely reflects a combination of genetic susceptibility and an unknown
environmental insult. Moreover, the identification of genes and proteins that
may cause hereditary parkinsonism substantially contributes to our ability to
understand the pathogenesis of Parkinson's disease and may help in the early
identification of the disease and its treatment. The discovery of alpha-synuclein
mutations in families with autosomal dominant Parkinson's disease sheds light on
its role in sporadic Parkinson's disease. It seems that this protein tends to
aggregate when the cellular milieu is altered [14-16]. The question as to the
exact changes that cause its deposition remains open. One of the major
possibilities is oxidative stress [16]. The role of these aggregates in neuronal
cell death is also still unclear. Transgenic mice expressing wild-type human
alpha-synuclein developed progressive accumulation of alpha-synuclein and
ubiquitin-immunoreactive inclusions in neurons in the neocortex, hippocampus and
the substantia nigra. These alterations were associated with loss of
dopaminergic terminals and motor impairments [24]. This finding suggests that
accumulation of alpha-synuclein may play a causal role in sporadic Parkinson's
disease as well. The parkin protein seems to be a crucial survival factor for
nigral neurons [15]. The parkin protein is related to the ubiquitin pathway,
which is important in the elimination of damaged proteins. Ubiquitin-mediated
degradation of proteins plays a central role in the control of numerous
processes, including signal transduction, receptor and transcriptional
regulations, programmed cell death, and breakdown of abnormal proteins that may
interfere with normal cell functions. Further studies on the function of Parkin
protein and its relation to the ubiquitin pathway could elucidate at least one
of the molecular mechanisms of nigral neuronal death. A mutation in the
ubiquitin carboxy-teminal hydrolase L1 gene also implies the importance of the
ubiquitin pathway in Parkinson's disease. Abnormal tau protein was found to be
the cause of familial frontotemporal dementia and parkinsonism. It tends to form
filamentous structures, which may lead to neuronal death. Elucidation of the
molecular mechanism of neuronal death in this disease may contribute to our
understanding of sporadic diseases with tau accumulation, such as corticobasal
degeneration, progressive supranuclear palsy, Pick's disease, Alzheimer's
disease and possibly also the pathogenesis of Parkinson's disease. Other genetic
loci have been identified by linkage analysis of patients with familial
parkinsonism. These loci conceal other genes and proteins that may be pivotal
factors in the pathogenesis of Parkinson's disease. The discovery of genetic
mutations in patients with parkinsonism may offer us new insights into the
understanding of the pathways leading to neuronal death and development of
Parkinson's disease. It may also help in the early identification of susceptible
people to this disease and possibly in developing new treatment strategies.
Liu, Z., Y. Wang, et al. (2001). "Peptide derived from insulin with regulatory
activity of dopamine transporter." Neuropharmacology41(4):
464-71.
A nonapeptide derived from the C terminus of the insulin B chain,
H(2)N-Arg-Gly-Phe-Phe-Tyr-Thr-Pro-Lys-Ala-COOH, was found to strongly inhibit
dopamine (DA) uptake by rat dopamine transporter (DAT) stably expressed in CHO
cells (designated D8 cells). The kinetic experiments on D8 cells gave a curve
typical of competitive inhibition with an IC(50)=6.9 microM. This inhibitory
effect was also confirmed by experiments on striatal synaptosomes. The rat
administered with the nonapeptide unilaterally into substantia nigra showed
dose-dependent velocity and duration of the round movement contralateral to the
nonapeptide-injected side. In addition, the nonapeptide dose-dependently reduced
the binding of the tritium-labeled cocaine analog (-)-2 beta-carbomethoxy-3
beta-(4-fluorophenyl)tropane (WIN35,428) to DAT of D8 cells, which suggests that
the nonapeptide may inhibit the transport activity of DAT in the way as cocaine
does. Meanwhile, the peptide DOI (insulin with 8 amino acid residues deleted at
the C terminus of the B chain) shows a significantly stimulating effect on DAT
uptake activity in D8 cells. So insulin is proposed as a kind of neuropeptide
precursor in the brain and insulin-derived peptides may be involved in the
process of regulating the DA system, and these peptides may be developed into
new medicines for disorders concerning the DA system such as Parkinson's disease
and cocaine addiction.
Shastry, B. S. (2001). "Parkinson disease: etiology, pathogenesis and future of
gene therapy." Neurosci Res41(1): 5-12.
Parkinson disease (PD) is a progressive neurological disorder with a prevalence
of 1-2% in people over the age of 50. It has a world-wide distribution and has
no gender preference. The neurological hallmark of PD is the presence of Lewy
bodies and is characterized by the degeneration of nigrostriatal dopaminergic
neurons. The causes of PD are unknown but considerable evidence suggests a
multifactorial etiology involving genetic and environmental factors. A molecular
genetic approach identified three genes and at least two additional loci in rare
familial forms of PD. Two of these genes are involved in the ubiquitin mediated
pathway of protein degradation and the third one is a highly expressed protein
in the synaptic terminal and is called alpha-synuclein. In animal models, it has
been shown that use of the household pesticide which is known to contain
rotenone, causes PD. Thus, a combined action of genetic and environmental
factors is responsible for the pathogenesis of PD. Although use of levodopa or
dopamine agonists can substantially reduce clinical symptoms, and
transplantation of fetal nerve tissue still remains as an alternative therapy
(although it has been recently shown to be having no overall benefit), directed
delivery of glial cell derived neurotrophic factor (known to have trophic
effects on dopaminergic neurons) may also be a beneficial therapeutic option for
PD patients.
Valente, E. M., A. R. Bentivoglio, et al. (2001). "Localization of a novel locus
for autosomal recessive early-onset parkinsonism, PARK6, on human chromosome
1p35-p36." Am J Hum Genet68(4): 895-900.
The cause of Parkinson disease (PD) is still unknown, but genetic factors have
recently been implicated in the etiology of the disease. So far, four loci
responsible for autosomal dominant PD have been identified. Autosomal recessive
juvenile parkinsonism (ARJP) is a clinically and genetically distinct entity;
typical PD features are associated with early onset, sustained response to
levodopa, and early occurrence of levodopa-induced dyskinesias, which are often
severe. To date, only one ARJP gene, Parkin, has been identified, and multiple
mutations have been detected both in families with autosomal recessive
parkinsonism and in sporadic cases. The Parkin-associated phenotype is broad,
and some cases are indistinguishable from idiopathic PD. In > or = 50% of
families with ARJP that have been analyzed, no mutations could be detected in
the Parkin gene. We identified a large Sicilian family with four definitely
affected members (the Marsala kindred). The phenotype was characterized by
early-onset (range 32-48 years) parkinsonism, with slow progression and
sustained response to levodopa. Linkage of the disease to the Parkin gene was
excluded. A genomewide homozygosity screen was performed in the family. Linkage
analysis and haplotype construction allowed identification of a single region of
homozygosity shared by all the affected members, spanning 12.5 cM on the short
arm of chromosome 1. This region contains a novel locus for autosomal recessive
early-onset parkinsonism, PARK6. A maximum LOD score 4.01 at recombination
fraction .00 was obtained for marker D1S199.
Vaughan, J. R., M. B. Davis, et al. (2001). "Genetics of Parkinsonism: a
review." Ann Hum Genet65(Pt 2): 111-26.
Idiopathic Parkinson's disease (IPD), a progressive neurodegenerative disorder,
is a common cause of disability. No current therapies modify disease
progression. The pathological hallmarks are the presence of Lewy bodies and
massive loss of dopaminergic neurons in the pars compacta of the substantia
nigra. Two genes (SNCA and parkin) as well as two gene loci have now been
implicated in the pathogenesis of familial PD. These represent significant
progress in our understanding of the disease, considering the rarity of large
families, low heritability in the general population and genetic heterogeneity.
Mutations in a further gene, UCHL1, have been described in familial PD although
the evidence for its role in PD is less clear. Knowledge of the genes described
in PD to date should help to define molecular mechanisms of neurodegeneration in
PD, as well as in other diseases where defects in protein handling may be a
common feature. Nigral degeneration with Lewy body formation and the resulting
clinical picture of PD may represent a final common pathway of a multifactorial
disease process in which both environmental and genetic factors have a role.
This review discusses the major advances in the field to date and illustrates
how the existence of genetic factors has now become firmly established.