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  • Acquired Immunodeficiency Syndrome/immunology  (1)
  • Dependovirus/genetics  (1)
  • 1
    Publication Date: 2004-11-06
    Description: Most mutations in the dystrophin gene create a frameshift or a stop in the mRNA and are associated with severe Duchenne muscular dystrophy. Exon skipping that naturally occurs at low frequency sometimes eliminates the mutation and leads to the production of a rescued protein. We have achieved persistent exon skipping that removes the mutated exon on the dystrophin messenger mRNA of the mdx mouse, by a single administration of an AAV vector expressing antisense sequences linked to a modified U7 small nuclear RNA. We report the sustained production of functional dystrophin at physiological levels in entire groups of muscles and the correction of the muscular dystrophy.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Goyenvalle, Aurelie -- Vulin, Adeline -- Fougerousse, Francoise -- Leturcq, France -- Kaplan, Jean-Claude -- Garcia, Luis -- Danos, Olivier -- New York, N.Y. -- Science. 2004 Dec 3;306(5702):1796-9. Epub 2004 Nov 4.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Genethon & CNRS UMR 8115, 1, rue de l'Internationale, Evry, France.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/15528407" target="_blank"〉PubMed〈/a〉
    Keywords: Animals ; Dependovirus/genetics ; Dystrophin/*genetics/metabolism ; *Exons ; *Genetic Therapy ; Genetic Vectors ; Introns ; Mice ; Mice, Inbred mdx ; Muscle Contraction ; Muscle Fibers, Skeletal/immunology/pathology ; Muscle, Skeletal/metabolism/pathology/physiology ; Muscular Dystrophy, Animal/genetics/pathology/physiopathology/*therapy ; Muscular Dystrophy, Duchenne/genetics/pathology/physiopathology/*therapy ; *Mutation ; Oligonucleotides, Antisense/*pharmacology ; RNA Splicing ; RNA, Messenger/genetics/metabolism ; RNA, Small Nuclear/genetics/*metabolism ; Transfection
    Print ISSN: 0036-8075
    Electronic ISSN: 1095-9203
    Topics: Biology , Chemistry and Pharmacology , Computer Science , Medicine , Natural Sciences in General , Physics
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  • 2
    Publication Date: 1988-02-26
    Description: Rabbit antisera were raised against three overlapping synthetic peptides with sequence homology to the second conserved domain of the external envelope glycoprotein (gp120) of the human immunodeficiency virus (HIV). All of the antisera immunoprecipitated the envelope glycoprotein. In particular, an antiserum directed against amino acids 254 to 274 of env was efficient in neutralizing three different isolates of HIV in vitro, without affecting the binding of the virus to CD4-positive cells. Therefore, this conserved region of gp120 appears to be critical in a postbinding event during virus penetration and may represent a target for antibody neutralization of HIV. These findings may be applicable in the design of a vaccine for the acquired immunodeficiency syndrome.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Ho, D D -- Kaplan, J C -- Rackauskas, I E -- Gurney, M E -- AI25541/AI/NIAID NIH HHS/ -- KO8-AI00685/AI/NIAID NIH HHS/ -- NS21442/NS/NINDS NIH HHS/ -- etc. -- New York, N.Y. -- Science. 1988 Feb 26;239(4843):1021-3.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Division of Infectious Diseases, Cedars-Sinai Medical Center, Los Angeles, CA.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/2830667" target="_blank"〉PubMed〈/a〉
    Keywords: Acquired Immunodeficiency Syndrome/immunology ; Amino Acid Sequence ; Antibodies, Viral/immunology ; Antigens, Differentiation, T-Lymphocyte/immunology ; Glucose-6-Phosphate Isomerase ; Growth Substances ; HIV/*immunology/physiology ; HIV Antibodies ; HIV Envelope Protein gp120 ; HIV Seropositivity ; Hemocyanin/immunology ; Humans ; Immune Sera/immunology ; Immunization ; Immunosorbent Techniques ; Lymphokines ; Molecular Sequence Data ; Neutralization Tests ; Receptors, Antigen, T-Cell/immunology ; Retroviridae Proteins/*immunology/physiology ; Sequence Homology, Nucleic Acid ; T-Lymphocytes/immunology/microbiology ; Viral Envelope Proteins/*immunology/physiology
    Print ISSN: 0036-8075
    Electronic ISSN: 1095-9203
    Topics: Biology , Chemistry and Pharmacology , Computer Science , Medicine , Natural Sciences in General , Physics
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