Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients
Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1–9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes t...
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| Published in | Current biology Vol. 19; no. 9; pp. 774 - 778 |
|---|---|
| Main Authors | , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
England
Elsevier Inc
12.05.2009
|
| Subjects | |
| Online Access | Get full text |
| ISSN | 0960-9822 1879-0445 1879-0445 |
| DOI | 10.1016/j.cub.2009.03.030 |
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| Abstract | Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs)
[1–9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the
Huntingtin gene, creating an extended polyglutamine tract that makes the Huntingtin protein toxic
[10]. Silencing mutant
Huntingtin messenger RNA (mRNA) should provide therapeutic benefit, but normal Huntingtin likely contributes to neuronal function
[11–13]. No siRNA strategy can yet distinguish among the normal and disease
Huntingtin alleles and other mRNAs containing CAG repeats
[14]. siRNAs targeting the disease isoform of a heterozygous single-nucleotide polymorphism (SNP) in
Huntingtin provide an alternative
[15–19]. We sequenced 22 predicted SNP sites in 225 human samples corresponding to HD and control subjects. We find that 48% of our patient population is heterozygous at a single SNP site; one isoform of this SNP is associated with HD. Several other SNP sites are frequently heterozygous. Consequently, five allele-specific siRNAs, corresponding to just three SNP sites, could be used to treat three-quarters of the United States and European HD patient populations. We have designed and validated selective siRNAs for the three SNP sites, laying the foundation for allele-specific RNA interference (RNAi) therapy for HD. |
|---|---|
| AbstractList | Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1-9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes the Huntingtin protein toxic [10]. Silencing mutant Huntingtin messenger RNA (mRNA) should provide therapeutic benefit, but normal Huntingtin likely contributes to neuronal function [11-13]. No siRNA strategy can yet distinguish among the normal and disease Huntingtin alleles and other mRNAs containing CAG repeats [14]. siRNAs targeting the disease isoform of a heterozygous single-nucleotide polymorphism (SNP) in Huntingtin provide an alternative [15-19]. We sequenced 22 predicted SNP sites in 225 human samples corresponding to HD and control subjects. We find that 48% of our patient population is heterozygous at a single SNP site; one isoform of this SNP is associated with HD. Several other SNP sites are frequently heterozygous. Consequently, five allele-specific siRNAs, corresponding to just three SNP sites, could be used to treat three-quarters of the United States and European HD patient populations. We have designed and validated selective siRNAs for the three SNP sites, laying the foundation for allele-specific RNA interference (RNAi) therapy for HD.Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1-9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes the Huntingtin protein toxic [10]. Silencing mutant Huntingtin messenger RNA (mRNA) should provide therapeutic benefit, but normal Huntingtin likely contributes to neuronal function [11-13]. No siRNA strategy can yet distinguish among the normal and disease Huntingtin alleles and other mRNAs containing CAG repeats [14]. siRNAs targeting the disease isoform of a heterozygous single-nucleotide polymorphism (SNP) in Huntingtin provide an alternative [15-19]. We sequenced 22 predicted SNP sites in 225 human samples corresponding to HD and control subjects. We find that 48% of our patient population is heterozygous at a single SNP site; one isoform of this SNP is associated with HD. Several other SNP sites are frequently heterozygous. Consequently, five allele-specific siRNAs, corresponding to just three SNP sites, could be used to treat three-quarters of the United States and European HD patient populations. We have designed and validated selective siRNAs for the three SNP sites, laying the foundation for allele-specific RNA interference (RNAi) therapy for HD. Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1–9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes the Huntingtin protein toxic [10]. Silencing mutant Huntingtin messenger RNA (mRNA) should provide therapeutic benefit, but normal Huntingtin likely contributes to neuronal function [11–13]. No siRNA strategy can yet distinguish among the normal and disease Huntingtin alleles and other mRNAs containing CAG repeats [14]. siRNAs targeting the disease isoform of a heterozygous single-nucleotide polymorphism (SNP) in Huntingtin provide an alternative [15–19]. We sequenced 22 predicted SNP sites in 225 human samples corresponding to HD and control subjects. We find that 48% of our patient population is heterozygous at a single SNP site; one isoform of this SNP is associated with HD. Several other SNP sites are frequently heterozygous. Consequently, five allele-specific siRNAs, corresponding to just three SNP sites, could be used to treat three-quarters of the United States and European HD patient populations. We have designed and validated selective siRNAs for the three SNP sites, laying the foundation for allele-specific RNA interference (RNAi) therapy for HD. Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) , and . Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes the Huntingtin protein toxic . Silencing mutant Huntingtin messenger RNA (mRNA) should provide therapeutic benefit, but normal Huntingtin likely contributes to neuronal function , and . No siRNA strategy can yet distinguish among the normal and disease Huntingtin alleles and other mRNAs containing CAG repeats . siRNAs targeting the disease isoform of a heterozygous single-nucleotide polymorphism (SNP) in Huntingtin provide an alternative , and . We sequenced 22 predicted SNP sites in 225 human samples corresponding to HD and control subjects. We find that 48% of our patient population is heterozygous at a single SNP site; one isoform of this SNP is associated with HD. Several other SNP sites are frequently heterozygous. Consequently, five allele-specific siRNAs, corresponding to just three SNP sites, could be used to treat three-quarters of the United States and European HD patient populations. We have designed and validated selective siRNAs for the three SNP sites, laying the foundation for allele-specific RNA interference (RNAi) therapy for HD. Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1-9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes the Huntingtin protein toxic [10]. Silencing mutant Huntingtin messenger RNA (mRNA) should provide therapeutic benefit, but normal Huntingtin likely contributes to neuronal function [11-13]. No siRNA strategy can yet distinguish among the normal and disease Huntingtin alleles and other mRNAs containing CAG repeats [14]. siRNAs targeting the disease isoform of a heterozygous single-nucleotide polymorphism (SNP) in Huntingtin provide an alternative [15-19]. We sequenced 22 predicted SNP sites in 225 human samples corresponding to HD and control subjects. We find that 48% of our patient population is heterozygous at a single SNP site; one isoform of this SNP is associated with HD. Several other SNP sites are frequently heterozygous. Consequently, five allele-specific siRNAs, corresponding to just three SNP sites, could be used to treat three-quarters of the United States and European HD patient populations. We have designed and validated selective siRNAs for the three SNP sites, laying the foundation for allele-specific RNA interference (RNAi) therapy for HD. |
| Author | Straubhaar, Juerg Vonsattel, Jean-Paul Liu, Wanzhou Pfister, Edith L. Wagh, Sujata Landwehrmeyer, Bernhard Kennington, Lori Aronin, Neil DiFiglia, Marian Zamore, Phillip D. |
| Author_xml | – sequence: 1 givenname: Edith L. surname: Pfister fullname: Pfister, Edith L. organization: Department of Medicine, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA – sequence: 2 givenname: Lori surname: Kennington fullname: Kennington, Lori organization: Department of Medicine, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA – sequence: 3 givenname: Juerg surname: Straubhaar fullname: Straubhaar, Juerg organization: Department of Medicine, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA – sequence: 4 givenname: Sujata surname: Wagh fullname: Wagh, Sujata organization: Department of Medicine, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA – sequence: 5 givenname: Wanzhou surname: Liu fullname: Liu, Wanzhou organization: Department of Medicine, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA – sequence: 6 givenname: Marian surname: DiFiglia fullname: DiFiglia, Marian organization: Massachusetts General Hospital, Charlestown, MA 02129, USA – sequence: 7 givenname: Bernhard surname: Landwehrmeyer fullname: Landwehrmeyer, Bernhard organization: University of Ulm, 89069 Ulm, Germany – sequence: 8 givenname: Jean-Paul surname: Vonsattel fullname: Vonsattel, Jean-Paul organization: Columbia University School of Medicine, New York, NY 10032, USA – sequence: 9 givenname: Phillip D. surname: Zamore fullname: Zamore, Phillip D. email: phillip.zamore@umassmed.edu organization: Department of Biochemistry and Molecular Biology, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA – sequence: 10 givenname: Neil surname: Aronin fullname: Aronin, Neil email: neil.aronin@umassmed.edu organization: Department of Medicine, Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA 01655, USA |
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[1–9].... Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1-9].... Among dominant neurodegenerative disorders, Huntington's disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) , and .... |
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| SubjectTerms | DNA Genetic Carrier Screening Genetic Therapy - methods Humans HUMDISEASE Huntingtin Protein Huntington Disease - genetics Huntington Disease - therapy Nerve Tissue Proteins - genetics Nuclear Proteins - genetics Polymorphism, Single Nucleotide - genetics RNA RNA Interference RNA, Small Interfering - genetics Sequence Analysis, DNA |
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| Title | Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients |
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