Gendaq Limited

Imperial College Innovations Limited

Gendaq Limited, C/o Brobeck Hale and Dorr

Cellectis

Fundacio Centre De Regulacio Genomica

Mark Isalan

Yen Choo

Aaron Klug

Xiaohai Liu

Michael Moore

Christopher Graeme Ullman

Sachin Dinesh Patel

John Girdlestone

François Stricher

Lindsey Reynolds

Christophe Demaison

Sylvestre Grizot

Shhankar Balasubramanian

Luis Serrano Pubul

Aaron Mrc Laboratory Of Molecular Biology Klug

Emmanuel Fajardo Sanchez

Yen Mrc Laboratory Of Molecular Biology Choo

Disclosed herein are novel nucleic acid molecules which encode novel polypeptides for use in treating polyglutamine diseases such as Huntington's disease. The polypeptides of the invention exhibit reduced immunotoxicity in vivo and may have utility in sustained (mid- or long-term) expression in vivo. Also disclosed are methods for the treatment of polyglutamine diseases, such as Huntington's disease and the use of nucleic acids and polypeptides of the invention in medical therapy. Gene therapy methods and compositions, such as AAV vectors for use in gene therapy methods are also disclosed.

Nucleic acids, peptides and methods

The invention relates to a zinc finger polypeptide library in which each polypeptide comprises more than one zinc finger which has been at least partially randomized, and to a set of zinc finger polypeptide libraries which encode overlapping zinc finger polypeptides, each polypeptide comprising more than one zinc finger which has been at least partially randomized, and which polypeptide may be assembled after selection to form a multifinger zinc finger polypeptide.

Nucleic acid binding polypeptide library

The invention provides a method for designing a nucleic acid binding protein of the Cys2-His2 zinc finger class capable of binding to a nucleic acid quadruplet in a target nucleic acid sequence, wherein binding to base 4 of the quadruplet by an alpha-helical zinc finger nucleic acid binding motif in the protein is determined as follows: if base 4 in the quadruplet is A, then position +6 in the alpha-helix is Glu, Asn or Val; if base 4 in the quadruplet is C, then position +6 in the alpha-helix is Ser, Thr, Val, Ala, Glu or Asn; if base 4 of the quadruplet is G, the position +6 in the alpha helix is Arg or Lys; if position 4 in the quadruplet is T, then position +6 in the alpha helix is Ser, Thr, Val or Lys.

Nucleic acid binding proteins

Disclosed herein are methods and compositions for modulating HIV infection by regulating expression of one or more receptors.

Modulation of HIV infection

The application provides methods of designing zinc finger binding polypeptides for binding to particular target sequences comprising overlapping nucleotide quadruplets.

Disclosed herein are methods for designing DNA binding proteins comprising a plurality of zinc fingers and methods for binding the proteins to target nucleotide sequences in cells.

The invention provides a method for producing a zinc finger polypeptide which binds to a target nucleic acid sequence containing a modified base but not to an identical sequence containing an equivalent unmodified base.

Disclosed herein are methods of designing zinc finger binding polypeptides for binding to particular target sequences comprising overlapping nucleotide quadruplets.

The invention provides a method for preparing a nucleic acid binding protein of the Cys2-His2 zinc finger class capable of binding to a nucleic acid quadruplet in a target nucleic acid sequence, wherein binding to base 4 of the quadruplet by an alpha-helical zinc finger nucleic acid binding motif in the protein is determined as follows: if base 4 in the quadruplet is A, then position &plus;6 in the alpha-helix is Glu, Asn or Val; if base 4 in the quadruplet is C, then position &plus;6 in the alpha-helix is Ser, Thr, Val, Ala, Glu or Asn.

The present invention relates to isolated or purified molecule(s) capable of binding to one or more of telomeric, G-quadruplex, or G-quartet nucleic acid(s).

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London, United Kingdom

Mark Isalan