Gentamicin-induced readthrough of stop codons in duchenne muscular dystrophy

Objective The objective of this study was to establish the feasibility of long‐term gentamicin dosing to achieve stop codon readthrough and produce full‐length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving t...

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Bibliographic Details
Published inAnnals of neurology Vol. 67; no. 6; pp. 771 - 780
Main Authors Malik, Vinod, Rodino-Klapac, Louise R., Viollet, Laurence, Wall, Cheryl, King, Wendy, Al-Dahhak, Roula, Lewis, Sarah, Shilling, Christopher J., Kota, Janaiah, Serrano-Munuera, Carmen, Hayes, John, Mahan, John D., Campbell, Katherine J., Banwell, Brenda, Dasouki, Majed, Watts, Victoria, Sivakumar, Kumaraswamy, Bien-Willner, Ricardo, Flanigan, Kevin M., Sahenk, Zarife, Barohn, Richard J., Walker, Christopher M., Mendell, Jerry R.
Format Journal Article
LanguageEnglish
Published Hoboken Wiley Subscription Services, Inc., A Wiley Company 01.06.2010
Wiley-Liss
Subjects
Adolescent
Audiometry - methods
Biological and medical sciences
Child
Child, Preschool
Codon, Terminator - drug effects
Codon, Terminator - genetics
Cohort Studies
Creatine Kinase - blood
Diseases of striated muscles. Neuromuscular diseases
Enzyme-Linked Immunosorbent Assay - methods
Gentamicins - therapeutic use
Humans
Medical sciences
Muscle Cells - pathology
Muscular Dystrophy, Duchenne - blood
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - pathology
Mutation - genetics
Neurology
Protein Synthesis Inhibitors - therapeutic use
T-Lymphocytes - drug effects
T-Lymphocytes - pathology
Time Factors
Neuromuscular diseases
Nervous system diseases
Codon
Duchenne muscular dystrophy
Gentamicin
Genetic disease
Online AccessGet full text

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Abstract Objective The objective of this study was to establish the feasibility of long‐term gentamicin dosing to achieve stop codon readthrough and produce full‐length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD). Methods Two DMD cohorts received 14‐day gentamicin (7.5mg/kg/day): Cohort 1 (n = 10) stop codon patients and Cohort 2 (n = 8) frameshift controls. Two additional stop codon DMD cohorts were gentamicin treated (7.5mg/kg) for 6 months: Cohort 3 (n = 12) dosed weekly and Cohort 4 (n = 4) dosed twice weekly. Pre‐ and post‐treatment biopsies were assessed for dystrophin levels, as were clinical outcomes. Results In the 14‐day study, serum creatine kinase (CK) dropped by 50%, which was not seen in frameshift DMD controls. After 6 months of gentamicin, dystrophin levels significantly increased (p = 0.027); the highest levels reached 13 to 15% of normal (1 in Cohort 3, and 2 in Cohort 4), accompanied by reduced serum CK favoring drug‐induced readthrough of stop codons. This was supported by stabilization of strength and a slight increase in forced vital capacity. Pretreatment stable transcripts predicted an increase of dystrophin after gentamicin. Readthrough efficiency was not affected by the stop codon or its surrounding fourth nucleotide. In 1 subject, antigen‐specific interferon‐γ enzyme‐linked immunospot assay detected an immunogenic dystrophin epitope. Interpretation The results support efforts to achieve drug‐induced mutation suppression of stop codons. The immunogenic epitope resulting from readthrough emphasizes the importance of monitoring T‐cell immunity during clinical studies that suppress stop codons. Similar principles apply to other molecular strategies, including exon skipping and gene therapy. ANN NEUROL 2010;67:771–780
AbstractList Objective The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD). Methods Two DMD cohorts received 14-day gentamicin (7.5mg/kg/day): Cohort 1 (n = 10) stop codon patients and Cohort 2 (n = 8) frameshift controls. Two additional stop codon DMD cohorts were gentamicin treated (7.5mg/kg) for 6 months: Cohort 3 (n = 12) dosed weekly and Cohort 4 (n = 4) dosed twice weekly. Pre- and post-treatment biopsies were assessed for dystrophin levels, as were clinical outcomes. Results In the 14-day study, serum creatine kinase (CK) dropped by 50%, which was not seen in frameshift DMD controls. After 6 months of gentamicin, dystrophin levels significantly increased (p = 0.027); the highest levels reached 13 to 15% of normal (1 in Cohort 3, and 2 in Cohort 4), accompanied by reduced serum CK favoring drug-induced readthrough of stop codons. This was supported by stabilization of strength and a slight increase in forced vital capacity. Pretreatment stable transcripts predicted an increase of dystrophin after gentamicin. Readthrough efficiency was not affected by the stop codon or its surrounding fourth nucleotide. In 1 subject, antigen-specific interferon- enzyme-linked immunospot assay detected an immunogenic dystrophin epitope. Interpretation The results support efforts to achieve drug-induced mutation suppression of stop codons. The immunogenic epitope resulting from readthrough emphasizes the importance of monitoring T-cell immunity during clinical studies that suppress stop codons. Similar principles apply to other molecular strategies, including exon skipping and gene therapy. ANN NEUROL 2010; 67:771-780.
The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD). Two DMD cohorts received 14-day gentamicin (7.5mg/kg/day): Cohort 1 (n = 10) stop codon patients and Cohort 2 (n = 8) frameshift controls. Two additional stop codon DMD cohorts were gentamicin treated (7.5mg/kg) for 6 months: Cohort 3 (n = 12) dosed weekly and Cohort 4 (n = 4) dosed twice weekly. Pre- and post-treatment biopsies were assessed for dystrophin levels, as were clinical outcomes. In the 14-day study, serum creatine kinase (CK) dropped by 50%, which was not seen in frameshift DMD controls. After 6 months of gentamicin, dystrophin levels significantly increased (p = 0.027); the highest levels reached 13 to 15% of normal (1 in Cohort 3, and 2 in Cohort 4), accompanied by reduced serum CK favoring drug-induced readthrough of stop codons. This was supported by stabilization of strength and a slight increase in forced vital capacity. Pretreatment stable transcripts predicted an increase of dystrophin after gentamicin. Readthrough efficiency was not affected by the stop codon or its surrounding fourth nucleotide. In 1 subject, antigen-specific interferon-gamma enzyme-linked immunospot assay detected an immunogenic dystrophin epitope. The results support efforts to achieve drug-induced mutation suppression of stop codons. The immunogenic epitope resulting from readthrough emphasizes the importance of monitoring T-cell immunity during clinical studies that suppress stop codons. Similar principles apply to other molecular strategies, including exon skipping and gene therapy.
Objective The objective of this study was to establish the feasibility of long‐term gentamicin dosing to achieve stop codon readthrough and produce full‐length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD). Methods Two DMD cohorts received 14‐day gentamicin (7.5mg/kg/day): Cohort 1 (n = 10) stop codon patients and Cohort 2 (n = 8) frameshift controls. Two additional stop codon DMD cohorts were gentamicin treated (7.5mg/kg) for 6 months: Cohort 3 (n = 12) dosed weekly and Cohort 4 (n = 4) dosed twice weekly. Pre‐ and post‐treatment biopsies were assessed for dystrophin levels, as were clinical outcomes. Results In the 14‐day study, serum creatine kinase (CK) dropped by 50%, which was not seen in frameshift DMD controls. After 6 months of gentamicin, dystrophin levels significantly increased (p = 0.027); the highest levels reached 13 to 15% of normal (1 in Cohort 3, and 2 in Cohort 4), accompanied by reduced serum CK favoring drug‐induced readthrough of stop codons. This was supported by stabilization of strength and a slight increase in forced vital capacity. Pretreatment stable transcripts predicted an increase of dystrophin after gentamicin. Readthrough efficiency was not affected by the stop codon or its surrounding fourth nucleotide. In 1 subject, antigen‐specific interferon‐γ enzyme‐linked immunospot assay detected an immunogenic dystrophin epitope. Interpretation The results support efforts to achieve drug‐induced mutation suppression of stop codons. The immunogenic epitope resulting from readthrough emphasizes the importance of monitoring T‐cell immunity during clinical studies that suppress stop codons. Similar principles apply to other molecular strategies, including exon skipping and gene therapy. ANN NEUROL 2010;67:771–780
The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD).OBJECTIVEThe objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD).Two DMD cohorts received 14-day gentamicin (7.5mg/kg/day): Cohort 1 (n = 10) stop codon patients and Cohort 2 (n = 8) frameshift controls. Two additional stop codon DMD cohorts were gentamicin treated (7.5mg/kg) for 6 months: Cohort 3 (n = 12) dosed weekly and Cohort 4 (n = 4) dosed twice weekly. Pre- and post-treatment biopsies were assessed for dystrophin levels, as were clinical outcomes.METHODSTwo DMD cohorts received 14-day gentamicin (7.5mg/kg/day): Cohort 1 (n = 10) stop codon patients and Cohort 2 (n = 8) frameshift controls. Two additional stop codon DMD cohorts were gentamicin treated (7.5mg/kg) for 6 months: Cohort 3 (n = 12) dosed weekly and Cohort 4 (n = 4) dosed twice weekly. Pre- and post-treatment biopsies were assessed for dystrophin levels, as were clinical outcomes.In the 14-day study, serum creatine kinase (CK) dropped by 50%, which was not seen in frameshift DMD controls. After 6 months of gentamicin, dystrophin levels significantly increased (p = 0.027); the highest levels reached 13 to 15% of normal (1 in Cohort 3, and 2 in Cohort 4), accompanied by reduced serum CK favoring drug-induced readthrough of stop codons. This was supported by stabilization of strength and a slight increase in forced vital capacity. Pretreatment stable transcripts predicted an increase of dystrophin after gentamicin. Readthrough efficiency was not affected by the stop codon or its surrounding fourth nucleotide. In 1 subject, antigen-specific interferon-gamma enzyme-linked immunospot assay detected an immunogenic dystrophin epitope.RESULTSIn the 14-day study, serum creatine kinase (CK) dropped by 50%, which was not seen in frameshift DMD controls. After 6 months of gentamicin, dystrophin levels significantly increased (p = 0.027); the highest levels reached 13 to 15% of normal (1 in Cohort 3, and 2 in Cohort 4), accompanied by reduced serum CK favoring drug-induced readthrough of stop codons. This was supported by stabilization of strength and a slight increase in forced vital capacity. Pretreatment stable transcripts predicted an increase of dystrophin after gentamicin. Readthrough efficiency was not affected by the stop codon or its surrounding fourth nucleotide. In 1 subject, antigen-specific interferon-gamma enzyme-linked immunospot assay detected an immunogenic dystrophin epitope.The results support efforts to achieve drug-induced mutation suppression of stop codons. The immunogenic epitope resulting from readthrough emphasizes the importance of monitoring T-cell immunity during clinical studies that suppress stop codons. Similar principles apply to other molecular strategies, including exon skipping and gene therapy.INTERPRETATIONThe results support efforts to achieve drug-induced mutation suppression of stop codons. The immunogenic epitope resulting from readthrough emphasizes the importance of monitoring T-cell immunity during clinical studies that suppress stop codons. Similar principles apply to other molecular strategies, including exon skipping and gene therapy.
Author Sahenk, Zarife
Mahan, John D.
Sivakumar, Kumaraswamy
Dasouki, Majed
Mendell, Jerry R.
Banwell, Brenda
Barohn, Richard J.
Watts, Victoria
Viollet, Laurence
Walker, Christopher M.
King, Wendy
Lewis, Sarah
Shilling, Christopher J.
Hayes, John
Al-Dahhak, Roula
Rodino-Klapac, Louise R.
Kota, Janaiah
Wall, Cheryl
Serrano-Munuera, Carmen
Flanigan, Kevin M.
Malik, Vinod
Bien-Willner, Ricardo
Campbell, Katherine J.
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  surname: Malik
  fullname: Malik, Vinod
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 2
  givenname: Louise R.
  surname: Rodino-Klapac
  fullname: Rodino-Klapac, Louise R.
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 3
  givenname: Laurence
  surname: Viollet
  fullname: Viollet, Laurence
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
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  givenname: Cheryl
  surname: Wall
  fullname: Wall, Cheryl
  organization: Department of Neurology, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
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  givenname: Wendy
  surname: King
  fullname: King, Wendy
  organization: Department of Neurology, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
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  fullname: Al-Dahhak, Roula
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  givenname: Sarah
  surname: Lewis
  fullname: Lewis, Sarah
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
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  givenname: Christopher J.
  surname: Shilling
  fullname: Shilling, Christopher J.
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 9
  givenname: Janaiah
  surname: Kota
  fullname: Kota, Janaiah
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
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  givenname: Carmen
  surname: Serrano-Munuera
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  organization: Department of Neurology, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 11
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  organization: Department of Pediatrics, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 12
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  surname: Mahan
  fullname: Mahan, John D.
  organization: Department of Pediatrics, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 13
  givenname: Katherine J.
  surname: Campbell
  fullname: Campbell, Katherine J.
  organization: Center for Vaccines and Immunity, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 14
  givenname: Brenda
  surname: Banwell
  fullname: Banwell, Brenda
  organization: Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
– sequence: 15
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  organization: Department of Neurology, University of Kansas, Kansas City, KS
– sequence: 16
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  fullname: Watts, Victoria
  organization: Department of Neurology, University of Kansas, Kansas City, KS
– sequence: 17
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  fullname: Sivakumar, Kumaraswamy
  organization: Neuromuscular Research Center, Scottsdale, AZ
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  organization: Neuromuscular Research Center, Scottsdale, AZ
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  givenname: Kevin M.
  surname: Flanigan
  fullname: Flanigan, Kevin M.
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
– sequence: 20
  givenname: Zarife
  surname: Sahenk
  fullname: Sahenk, Zarife
  organization: Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Ohio State University, Columbus, OH
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  surname: Barohn
  fullname: Barohn, Richard J.
  organization: Department of Neurology, University of Kansas, Kansas City, KS
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https://www.ncbi.nlm.nih.gov/pubmed/20517938$$D View this record in MEDLINE/PubMed
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Keywords Neuromuscular diseases
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Codon
Duchenne muscular dystrophy
Gentamicin
Genetic disease
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2007; 17
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2004; 10
2007; 357
1987; 44
2007; 117
1989; 320
1996; 2
2001; 57
2005; 11
1964; 51
1998; 33
2007; 47
2003; 22
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References_xml – reference: van Deutekom JC, Janson AA, Ginjaar LB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007; 357: 2677-2686.
– reference: Canton R, Cobos N, de Gracia J, et al. Antimicrobial therapy for pulmonary pathogenic colonisation and infection by Pseudomonas aeruginosa in cystic fibrosis patients. Clin Microbiol Infect 2005; 11: 690-703.
– reference: Disset A, Bourgeois CF, Benmalek N, et al. An exon skipping-associated nonsense mutation in the dystrophin gene uncovers a complex interplay between multiple antagonistic splicing elements. Hum Mol Genet 2006; 15: 999-1013.
– reference: Selimoglu E. Aminoglycoside-induced ototoxicity. Curr Pharm Des 2007; 13: 119-126.
– reference: Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve 1983; 6: 91-103.
– reference: Gurtler N, Schmuziger N, Kim Y, et al. Audiologic testing and molecular analysis of 12S rRNA in patients receiving aminoglycosides. Laryngoscope 2005; 115: 640-644.
– reference: Schubert MC, Tusa RJ, Grine LE, Herdman SJ. Optimizing the sensitivity of the head thrust test for identifying vestibular hypofunction. Phys Ther 2004; 84: 151-158.
– reference: DelloRusso C, Scott JM, Hartigan-O'Connor D, et al. Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc Natl Acad Sci U S A 2002; 99: 12979-12984.
– reference: Mendell JR, Campbell K, Rodino-Klapac L, et al. Immunity to dystrophin revealed by gene therapy for Duchenne muscular dystrophy. N Engl J Med. In press.
– reference: Tang HY, Hutcheson E, Neill S, et al. Genetic susceptibility to aminoglycoside ototoxicity: how many are at risk? Genet Med 2002; 4: 336-345.
– reference: Politano L, Nigro G, Nigro V, et al. Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results. Acta Myol 2003; 22: 15-21.
– reference: Wells DJ, Wells KE, Asante EA, et al. Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy. Hum Mol Genet 1995; 4: 1245-1250.
– reference: Flanigan KM, von Niederhausern A, Dunn DM, et al. Rapid direct sequence analysis of the dystrophin gene. Am J Hum Genet 2003; 72: 931-939.
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Snippet Objective The objective of this study was to establish the feasibility of long‐term gentamicin dosing to achieve stop codon readthrough and produce full‐length...
The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length...
Objective The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length...
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SubjectTerms Adolescent
Audiometry - methods
Biological and medical sciences
Child
Child, Preschool
Codon, Terminator - drug effects
Codon, Terminator - genetics
Cohort Studies
Creatine Kinase - blood
Diseases of striated muscles. Neuromuscular diseases
Enzyme-Linked Immunosorbent Assay - methods
Gentamicins - therapeutic use
Humans
Medical sciences
Muscle Cells - pathology
Muscular Dystrophy, Duchenne - blood
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - pathology
Mutation - genetics
Neurology
Protein Synthesis Inhibitors - therapeutic use
T-Lymphocytes - drug effects
T-Lymphocytes - pathology
Time Factors
Title Gentamicin-induced readthrough of stop codons in duchenne muscular dystrophy
URI https://api.istex.fr/ark:/67375/WNG-SDP96Q4G-C/fulltext.pdf
https://onlinelibrary.wiley.com/doi/abs/10.1002%2Fana.22024
https://www.ncbi.nlm.nih.gov/pubmed/20517938
https://www.proquest.com/docview/733127899
https://www.proquest.com/docview/754550373
Volume 67
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