DEVICES AND METHODS FOR DELIVERING POLYNUCLEOTIDES INTO RETINAL CELLS OF THE MACULA AND FOVEA
First Claim
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1. A system for subretinal delivery of a vector to an eye of a human, comprising:
- (a) a fine-bore cannula, wherein the fine bore cannula is 27 to 45 gauge;
(b) a syringe; and
(c) greater than about 0.8 ml of a suspension comprising an effective amount of the vector;
wherein the vector comprises a polynucleotide encoding a therapeutic polypeptide or therapeutic RNA under the control of a promoter suitable for expression of the therapeutic polypeptide or therapeutic RNA in one or more central retina cell types; and
wherein the vector is useful for treatment of an ocular disorder when administered to the subretinal space of the central retina of the eye.
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Abstract
Methods and systems for the delivery of polynucleotides to the subretinal space of the macula or fovea of an eye of a human are provided. The methods and systems are useful for treating ocular disorders.
50 Citations
130 Claims
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1. A system for subretinal delivery of a vector to an eye of a human, comprising:
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(a) a fine-bore cannula, wherein the fine bore cannula is 27 to 45 gauge; (b) a syringe; and (c) greater than about 0.8 ml of a suspension comprising an effective amount of the vector; wherein the vector comprises a polynucleotide encoding a therapeutic polypeptide or therapeutic RNA under the control of a promoter suitable for expression of the therapeutic polypeptide or therapeutic RNA in one or more central retina cell types; and wherein the vector is useful for treatment of an ocular disorder when administered to the subretinal space of the central retina of the eye. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44, 45, 127, 128)
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46. A system for subretinal delivery of a vector to an eye of a human, comprising:
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(a) a fine-bore cannula, wherein the fine bore cannula is 27 to 45 gauge; (b) a first syringe comprising a first fluid suitable for subretinal injection to the eye; and (c) a second syringe comprising a second fluid comprising an effective amount of the vector; wherein the total volume of the first and the second fluids in combination is about 0.5 ml to about 3.0 ml; wherein the vector comprises a polynucleotide encoding a therapeutic polypeptide or therapeutic RNA under the control of a promoter suitable for expression of the therapeutic polypeptide or therapeutic RNA in one or more central retina cell types; and wherein the vector is useful for treatment of an ocular disorder when administered to the subretinal space of the central retina of the eye. - View Dependent Claims (47, 48, 49, 50, 51, 52, 53, 54, 55, 56, 57, 58, 59, 60, 61, 62, 63, 64, 65, 66, 67, 68, 69, 70, 71, 72, 73, 74, 75, 76, 77, 78, 79, 80, 81, 82, 83, 84, 85, 129, 130)
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86. A method for treating an ocular disorder, comprising:
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administering to the subretinal space of the central retina in an eye of a human in need thereof an effective amount of a vector; wherein the vector is useful for treatment of the ocular disorder when administered to the subretinal space of the central retina of the eye. - View Dependent Claims (87, 88, 89, 90, 91, 92, 93, 94, 95, 96, 97, 98, 99, 100, 101, 102, 103, 104, 105, 106, 107, 108, 109, 110, 111, 112, 113, 114, 115, 116, 117, 118, 119, 120, 121, 122, 123, 124, 125, 126)
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Specification