Dual phase - PNA conjugates for the delivery of PNA through the blood brain barrier
First Claim
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1. A molecule represented by any one of the formulas I-IV:
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[(L)t-(N)r-(H)q-(P)s]x
I.
[(L)t-(H)q-(N)r-(P)s]x
II.
[(P)s-(N)r-(H)q-(L)t]x
III.
[(P)s-(H)q-(N)r-(L)t]x
IV.wherein N is a nucleic acid sequence in a length of 1-100 bases, L is a peptide ligand which binds to a specific receptor and P is a positively charge moiety and H is a hydrophobic moiety; and
wherein r is an integer of 1-25, t is an integer of 1-50, s is an integer of 1-25, q is an integer of 0-20, and x is an integer of 1-20.
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Abstract
The invention provides molecule comprising a nucleic acid, a peptide ligand, which binds to a specific receptor and a positively charge peptide moiety with lysosomatic properties, useful in the delivery of a nucleic acid across a cellular membrane. The invention further relates to the use of these compounds for the delivery of a nucleic acid to the brain across the blood brain barrier for diagnostic and therapeutic applications
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Citations
58 Claims
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1. A molecule represented by any one of the formulas I-IV:
[(L)t-(N)r-(H)q-(P)s]x
I.
[(L)t-(H)q-(N)r-(P)s]x
II.
[(P)s-(N)r-(H)q-(L)t]x
III.
[(P)s-(H)q-(N)r-(L)t]x
IV.wherein N is a nucleic acid sequence in a length of 1-100 bases, L is a peptide ligand which binds to a specific receptor and P is a positively charge moiety and H is a hydrophobic moiety; and
wherein r is an integer of 1-25, t is an integer of 1-50, s is an integer of 1-25, q is an integer of 0-20, and x is an integer of 1-20. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 43, 45, 47, 49, 51, 52, 57)
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23. A molecule represented by any one of the formulas V-VIII:
[(L)t-(PNA)r-(H)q-(P)s]x
V.
[(L)t-(H)q-(PNA)r-(P)s]x
VI.
[(P)s-(PNA)r-(H)q-(L)t]x
VII.
[(P)s-(H)q-(PNA)r-(L)t]x
VIII.wherein the length of said PNA sequence is 1-100 bases, L is a peptide ligand which binds to a specific receptor and P is a positively charge moiety, and H is a hydrophobic moiety; and
wherein r is an integer of 1-25, t is an integer of 1-50, s is an integer of 0-25, q is an integer of 0-20 and x is an integer of 1-20. - View Dependent Claims (24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 44, 46, 48, 50, 53, 54, 56, 58)
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55. A method for delivering a nucleic acid sequence to the brain across the blood brain barrier, said method comprising the step of administering to a subject an effective amount of one or more molecules according to claim.
Specification