JPWO2019143803A5 - - Google Patents
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- JPWO2019143803A5 JPWO2019143803A5 JP2020537156A JP2020537156A JPWO2019143803A5 JP WO2019143803 A5 JPWO2019143803 A5 JP WO2019143803A5 JP 2020537156 A JP2020537156 A JP 2020537156A JP 2020537156 A JP2020537156 A JP 2020537156A JP WO2019143803 A5 JPWO2019143803 A5 JP WO2019143803A5
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- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 21
- 102000004169 proteins and genes Human genes 0.000 claims description 20
- 108090000623 proteins and genes Proteins 0.000 claims description 20
- 239000002245 particle Substances 0.000 claims description 16
- 108020004707 nucleic acids Proteins 0.000 claims description 9
- 150000007523 nucleic acids Chemical class 0.000 claims description 9
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 102100011550 ACTB Human genes 0.000 claims description 8
- 101700033661 ACTB Proteins 0.000 claims description 8
- 101710032514 ACTI Proteins 0.000 claims description 8
- 241000701022 Cytomegalovirus Species 0.000 claims description 8
- 230000001919 adrenal Effects 0.000 claims description 8
- 210000004027 cells Anatomy 0.000 claims description 8
- 102000014169 Steroid 21-Hydroxylase Human genes 0.000 claims description 7
- 108010011732 Steroid 21-Hydroxylase Proteins 0.000 claims description 7
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 6
- 210000002556 adrenal cortex cell Anatomy 0.000 claims description 6
- 238000002347 injection Methods 0.000 claims description 6
- 239000007924 injection Substances 0.000 claims description 6
- 241000432074 Adeno-associated virus Species 0.000 claims description 4
- 208000005676 Adrenogenital Syndrome Diseases 0.000 claims description 4
- 102000004040 Capsid Proteins Human genes 0.000 claims description 4
- 108090000565 Capsid Proteins Proteins 0.000 claims description 4
- 208000008448 Congenital Adrenal Hyperplasia Diseases 0.000 claims description 4
- 238000004519 manufacturing process Methods 0.000 claims description 4
- 210000000130 stem cell Anatomy 0.000 claims description 4
- 210000004404 Adrenal Cortex Anatomy 0.000 claims description 3
- 210000004100 Adrenal Glands Anatomy 0.000 claims description 3
- 229920000970 Repeated sequence (DNA) Polymers 0.000 claims description 3
- 210000001943 Adrenal Medulla Anatomy 0.000 claims description 2
- 210000001367 Arteries Anatomy 0.000 claims description 2
- 102100012498 CYP21A2 Human genes 0.000 claims description 2
- 101710037818 CYP21A2 Proteins 0.000 claims description 2
- 229920002676 Complementary DNA Polymers 0.000 claims description 2
- 210000004185 Liver Anatomy 0.000 claims description 2
- 210000001672 Ovary Anatomy 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 2
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 239000003085 diluting agent Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000002357 laparoscopic surgery Methods 0.000 claims description 2
- 238000004806 packaging method and process Methods 0.000 claims description 2
- 238000001356 surgical procedure Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 5
- 201000010099 disease Diseases 0.000 claims 2
- 210000002966 Serum Anatomy 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 206010000021 21-hydroxylase deficiency Diseases 0.000 description 3
- 208000009323 Congenital adrenal hyperplasia due to 21 hydroxylase deficiency Diseases 0.000 description 3
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
Description
本発明はまた、２１－ヒドロキシラーゼ欠損症を治療するための医薬の製造における、少なくとも１つのＡＡＶ逆末端反復配列（ＩＴＲ）および２１－ヒドロキシラーゼ（２１ＯＨ）タンパク質をコードする非ＡＡＶヌクレオチド配列を含む核酸分子を含むｒＡＡＶベクターまたはそのようなベクターを含むｒＡＡＶ粒子の使用であって、非ＡＡＶヌクレオチド配列がプロモーターに作動可能に連結されている、使用を想定する。
特定の実施形態では、例えば、以下が提供される：
（項目１）
少なくとも１つのＡＡＶ逆末端反復配列（ＩＴＲ）および２１－ヒドロキシラーゼ（２１ＯＨ）タンパク質をコードする非ＡＡＶヌクレオチド配列を含む核酸分子を含む組換えアデノ随伴ウイルス（ｒＡＡＶ）ベクターであって、前記非ＡＡＶヌクレオチド配列がプロモーターに作動可能に連結されている、ｒＡＡＶベクター。
（項目２）
前記２１ＯＨタンパク質がヒト２１ＯＨタンパク質である、項目１に記載のｒＡＡＶベクター。
（項目３）
２１ＯＨタンパク質をコードする前記非ＡＡＶヌクレオチド配列がヒト２１ＯＨ（ＣＹＰ２１Ａ２）ｃＤＮＡを含むまたはからなる、項目１に記載のｒＡＡＶベクター。
（項目４）
２１ＯＨタンパク質をコードする前記非ＡＡＶヌクレオチド配列がコドン最適化ヌクレオチド配列を含むまたはからなる、項目３に記載のｒＡＡＶベクター。
（項目５）
２１ＯＨタンパク質をコードする前記非ＡＡＶヌクレオチド配列が配列番号：５０を含むまたはからなる、項目１～４のいずれか１項に記載のｒＡＡＶベクター。
（項目６）
２１ＯＨタンパク質をコードする前記非ＡＡＶヌクレオチド配列が、配列番号：１のアミノ酸配列または配列番号：１に対して少なくとも９５％、少なくとも９６％、少なくとも９７％、少なくとも９８％もしくは少なくとも９９％同一のアミノ酸配列をコードする、項目１～５のいずれか１項に記載のｒＡＡＶベクター。
（項目７）
前記プロモーターが宿主細胞中での前記２１ＯＨタンパク質の発現を指令する、項目１～６のいずれか１項に記載のｒＡＡＶベクター。
（項目８）
前記宿主細胞が副腎細胞または副腎皮質細胞である、項目７に記載のｒＡＡＶベクター。
（項目９）
前記プロモーターが、サイトメガロウイルス／β－アクチンハイブリッドプロモーター、ＰＧＫプロモーターまたは発現が副腎皮質細胞において特異的なプロモーターである、項目１～８のいずれか１項に記載のｒＡＡＶベクター。
（項目１０）
前記サイトメガロウイルス／β－アクチンハイブリッドプロモーターが、ＣＡＧ、ＣＢ６またはＣＢＡプロモーターである、項目９に記載のｒＡＡＶベクター。
（項目１１）
前記プロモーターが、配列番号：２、配列番号：３、配列番号：４８または配列番号：４９のヌクレオチド配列を含むまたはからなる、項目１～１０のいずれか１項に記載のｒＡＡＶベクター。
（項目１２）
前記ＩＴＲが、ＡＡＶ１、ＡＡＶ２、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ９、ＡＡＶ１０、ＡＡＶ１１、ＡＡＶ１２、ｒｈ１０またはｒｈ７４血清型ＩＴＲである、項目１～１１のいずれか１項に記載のｒＡＡＶベクター。
（項目１３）
前記ｒＡＡＶが、ＡＡＶ１、ＡＡＶ２、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ９、ＡＡＶ１０、ＡＡＶ１１、ＡＡＶ１２、ｒｈ１０またはｒｈ７４血清型である、項目１～１２のいずれか１項に記載のｒＡＡＶベクター。
（項目１４）
２１－ヒドロキシラーゼ（２１ＯＨ）タンパク質をコードする非ＡＡＶヌクレオチド配列を含む核酸分子を含む組換えアデノ随伴ウイルス（ｒＡＡＶ）ベクターであって、前記非ＡＡＶヌクレオチド配列がプロモーターに作動可能に連結されており、
前記ｒＡＡＶベクターが少なくとも１つのＡＡＶ逆末端反復配列（ＩＴＲ）を含み、前記ＩＴＲが、血清型ＡＡＶ１、ＡＡＶ２、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ９、ＡＡＶ１０、ＡＡＶ１１、ＡＡＶ１２、ｒｈ１０またはｒｈ７４のＡＡＶからのものであり、かつ
前記プロモーターが、サイトメガロウイルス／β－アクチンハイブリッドプロモーター、ＰＧＫプロモーターまたは発現が副腎皮質細胞において特異的なプロモーターである、ｒＡＡＶベクター。
（項目１５）
前記サイトメガロウイルス／β－アクチンハイブリッドプロモーターが、ＣＡＧ、ＣＢ６またはＣＢＡプロモーターである、項目１４に記載のｒＡＡＶベクター。
（項目１６）
項目１～１５のいずれか１項に記載のｒＡＡＶベクターを含むｒＡＡＶ粒子。
（項目１７）
ＡＡＶ血清型ＡＡＶ１、ＡＡＶ２、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ９、ＡＡＶ１０、ＡＡＶ１１、ＡＡＶ１２、ｒｈ１０またはｒｈ７４からの少なくとも１つのキャプシドタンパク質をさらに含む、項目１６に記載のｒＡＡＶ粒子。
（項目１８）
項目１～１５のいずれか１項に記載のｒＡＡＶベクターまたは項目１６もしくは１７に記載のｒＡＡＶ粒子、および薬学的に許容される担体、希釈剤または賦形剤を含む、医薬組成物。
（項目１９）
ｒＡＡＶ粒子を製造する方法であって、（ａ）項目１～１５のいずれか１項に記載のｒＡＡＶベクター、（ｂ）ＡＡＶ ｒｅｐをコードする核酸分子、（ｃ）少なくとも１つのＡＡＶキャプシドタンパク質をコードする核酸分子および（ｄ）前記ｒＡＡＶ粒子をパッケージングするための充分なヘルパー機能を含有する宿主細胞を培養することを含む、方法。
（項目２０）
それを必要とする対象において２１－ヒドロキシラーゼ（２１ＯＨ）を発現させる方法であって、前記対象に治療有効量の、項目１～１５のいずれか１項に記載のｒＡＡＶベクター、項目１６もしくは１７に記載のｒＡＡＶ粒子または項目１８に記載の医薬組成物を投与し、それにより、前記対象において２１ＯＨを発現させることを含む、方法。
（項目２１）
前記２１ＯＨが、前記対象の副腎皮質、副腎髄質、副腎幹細胞、副腎前駆細胞、肝臓または卵巣中で発現される、項目２０に記載の方法。
（項目２２）
２１－ヒドロキシラーゼ欠損症（２１ＯＨＤ）を有する対象を治療する方法であって、前記対象に治療有効量の、項目１～１５のいずれか１項に記載のｒＡＡＶベクター、項目１６もしくは１７に記載のｒＡＡＶ粒子または項目１８に記載の医薬組成物を投与し、それにより、前記対象において２１ＯＨＤを治療することを含む、方法。
（項目２３）
前記投与するステップの前に２１ＯＨＤを有する対象を選択することをさらに含む、項目２２に記載の方法。
（項目２４）
前記ｒＡＡＶベクター、前記ｒＡＡＶ粒子または前記医薬組成物が、静脈内に、直視下手術もしくは腹腔鏡検査法を介して副腎への直接注射によりまたはカテーテル法を介して副腎動脈への注射により前記対象に投与される、項目１９～２３のいずれか１項に記載の方法。
（項目２５）
副腎への前記直接注射が副腎皮質への直接注射である、項目２４に記載の方法。
（項目２６）
前記対象が、プラダーのステージＩＶまたはＶの形態の２１ＯＨＤを罹患している、項目２２～２５のいずれか１項に記載の方法。
（項目２７）
前記対象が、先天性副腎過形成症（ＣＡＨ）を罹患している、項目２２～２６のいずれか１項に記載の方法。
（項目２８）
２１－ヒドロキシラーゼ欠損症を治療するための医薬の製造における、項目１～１５のいずれか１項に記載のｒＡＡＶベクターまたは項目１６もしくは１７に記載のｒＡＡＶ粒子の使用。
The invention also comprises a non-AAV nucleotide sequence encoding at least one AAV reverse-ended repeat (ITR) and 21-hydroxylase (21OH) protein in the manufacture of a pharmaceutical for treating 21-hydroxylase deficiency. It is envisioned the use of an rAAV vector comprising a nucleic acid molecule or an rAAV particle comprising such a vector, wherein the non-AAV nucleotide sequence is operably linked to a promoter.
In certain embodiments, for example, the following is provided:
(Item 1)
A recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid molecule comprising at least one AAV reverse-ended repeat sequence (ITR) and a non-AAV nucleotide sequence encoding a 21-hydroxylase (21OH) protein, said non-AAV nucleotide. An rAAV vector in which the sequence is operably linked to a promoter.
(Item 2)
The rAAV vector according to item 1, wherein the 21OH protein is a human 21OH protein.
(Item 3)
The rAAV vector according to item 1, wherein the non-AAV nucleotide sequence encoding a 21OH protein comprises or consists of human 21OH (CYP21A2) cDNA.
(Item 4)
The rAAV vector according to item 3, wherein the non-AAV nucleotide sequence encoding a 21OH protein comprises or consists of a codon-optimized nucleotide sequence.
(Item 5)
The rAAV vector according to any one of Items 1 to 4, wherein the non-AAV nucleotide sequence encoding a 21OH protein comprises or comprises SEQ ID NO: 50.
(Item 6)
The non-AAV nucleotide sequence encoding the 21OH protein is at least 95%, at least 96%, at least 97%, at least 98% or at least 99% identical to the amino acid sequence of SEQ ID NO: 1 or SEQ ID NO: 1. The rAAV vector according to any one of items 1 to 5, which encodes.
(Item 7)
The rAAV vector according to any one of items 1 to 6, wherein the promoter directs the expression of the 21OH protein in a host cell.
(Item 8)
The rAAV vector according to item 7, wherein the host cell is an adrenal cell or an adrenocortical cell.
(Item 9)
Item 6. The rAAV vector according to any one of Items 1 to 8, wherein the promoter is a cytomegalovirus / β-actin hybrid promoter, a PGK promoter or a promoter whose expression is specific in adrenocortical cells.
(Item 10)
9. The rAAV vector according to item 9, wherein the cytomegalovirus / β-actin hybrid promoter is a CAG, CB6 or CBA promoter.
(Item 11)
The rAAV vector according to any one of Items 1 to 10, wherein the promoter comprises or comprises the nucleotide sequence of SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 48 or SEQ ID NO: 49.
(Item 12)
The rAAV according to any one of items 1 to 11, wherein the ITR is AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, rh10 or rh74 serotype ITR. vector.
(Item 13)
The rAAV vector according to any one of items 1 to 12, wherein the rAAV is an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, rh10 or rh74 serotype. ..
(Item 14)
A recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid molecule comprising a non-AAV nucleotide sequence encoding a 21-hydroxylase (21OH) protein, wherein the non-AAV nucleotide sequence is operably linked to a promoter.
The rAAV vector comprises at least one AAV reverse terminal repeat sequence (ITR), wherein the ITR is a serotype AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, rh10 or It is from the AAV of rh74 and
The rAAV vector, wherein the promoter is a cytomegalovirus / β-actin hybrid promoter, a PGK promoter or a promoter whose expression is specific in adrenocortical cells.
(Item 15)
The rAAV vector according to item 14, wherein the cytomegalovirus / β-actin hybrid promoter is a CAG, CB6 or CBA promoter.
(Item 16)
The rAAV particle containing the rAAV vector according to any one of items 1 to 15.
(Item 17)
The rAAV particle according to item 16, further comprising at least one capsid protein from the AAV serotypes AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, rh10 or rh74.
(Item 18)
A pharmaceutical composition comprising the rAAV vector according to any one of items 1 to 15 or the rAAV particles according to item 16 or 17, and a pharmaceutically acceptable carrier, diluent or excipient.
(Item 19)
A method for producing rAAV particles, wherein (a) the rAAV vector according to any one of items 1 to 15, (b) a nucleic acid molecule encoding AAV rep, and (c) at least one AAV capsid protein are encoded. A method comprising culturing a host cell containing a nucleic acid molecule and (d) sufficient helper function for packaging the rAAV particles.
(Item 20)
A method for expressing 21-hydroxylase (21OH) in a subject in need thereof, wherein a therapeutically effective amount of the subject is the rAAV vector according to any one of items 1 to 15, item 16 or 17. A method comprising administering the described rAAV particles or the pharmaceutical composition according to item 18, thereby expressing 21OH in the subject.
(Item 21)
The method according to item 20, wherein the 21OH is expressed in the adrenal cortex, adrenal medulla, adrenal stem cells, adrenal progenitor cells, liver or ovary of the subject.
(Item 22)
A method for treating a subject having 21-hydroxylase deficiency (21OHD), wherein a therapeutically effective amount of the subject is the rAAV vector according to any one of items 1 to 15, item 16 or 17. A method comprising administering rAAV particles or the pharmaceutical composition according to item 18, thereby treating 21 OHD in the subject.
(Item 23)
22. The method of item 22, further comprising selecting a subject having 21 OHD prior to the dosing step.
(Item 24)
The rAAV vector, the rAAV particles or the pharmaceutical composition is administered intravenously to the subject by direct injection into the adrenal gland via direct surgery or laparoscopic surgery or by injection into the adrenal artery via catheterization. The method according to any one of items 19 to 23, which is administered.
(Item 25)
24. The method of item 24, wherein the direct injection into the adrenal gland is a direct injection into the adrenal cortex.
(Item 26)
The method of any one of items 22-25, wherein the subject suffers from 21OHD in the form of stage IV or V of Prader.
(Item 27)
The method according to any one of items 22 to 26, wherein the subject suffers from congenital adrenal hyperplasia (CAH).
(Item 28)
Use of the rAAV vector according to any one of items 1 to 15 or the rAAV particles according to item 16 or 17 in the manufacture of a pharmaceutical for treating 21-hydroxylase deficiency.
Claims (22)
（ｉ）前記非ＡＡＶヌクレオチド配列がプロモーターに作動可能に連結されており、前記プロモーターが、サイトメガロウイルス／β－アクチンハイブリッドプロモーター、ＰＧＫプロモーターまたは発現が副腎皮質細胞において特異的なプロモーターである、
（ｉｉ）前記ｒＡＡＶベクターが少なくとも１つのＡＡＶ逆末端反復配列（ＩＴＲ）を含み、前記ＩＴＲが、血清型ＡＡＶ１、ＡＡＶ２、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ９、ＡＡＶ１０、ＡＡＶ１１、ＡＡＶ１２、ｒｈ１０、またはｒｈ７４のＡＡＶからのものであり、かつ
（ｉｉｉ）前記ｒＡＡＶが、ＡＡＶ１、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ１０、ＡＡＶ１１、ＡＡＶ１２、またはｒｈ７４血清型である、ｒＡＡＶベクター。 A recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid molecule comprising a non-AAV nucleotide sequence encoding a 21-hydroxylase (21OH) protein.
(I) The non-AAV nucleotide sequence is operably linked to a promoter, which is a cytomegalovirus / β-actin hybrid promoter, PGK promoter or promoter specific for expression in adrenal cortex cells.
(Ii) The rAAV vector comprises at least one AAV reverse terminal repeat sequence (ITR), wherein the ITR contains serotypes AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12. , Rh10 , or rh74 from AAV, and
(Iii) An rAAV vector in which the rAAV is AAV1, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV10, AAV11, AAV12, or rh74 serotype .
（ｉ）前記対象の副腎皮質、副腎髄質、副腎幹細胞、副腎前駆細胞、肝臓、または卵巣中で２１ＯＨを発現するように、あるいは
（ｉｉ）前記対象への静脈内投与、直視下手術もしくは腹腔鏡検査法を介した前記対象の副腎への直接注射、またはカテーテル法を介した前記対象の副腎動脈への注射のために、
配合される、請求項２０に記載の組成物または医薬組成物。 The composition or the pharmaceutical composition
(I) to express 21OH in the adrenal cortex, adrenal medulla, adrenal stem cells, adrenal progenitor cells, liver, or ovary of the subject, or
(Ii) For intravenous administration to the subject, direct injection into the subject's adrenal gland via direct surgery or laparoscopic surgery , or injection into the subject's adrenal artery via catheterization .
The composition or pharmaceutical composition according to claim 20 , which is blended .
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| JP2023062403A JP2023076698A (en) | 2018-01-17 | 2023-04-06 | Adeno-associated virus gene therapy for 21-hydroxylase deficiency |
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| US201862618307P | 2018-01-17 | 2018-01-17 | |
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| US62/640,311 | 2018-03-08 | ||
| PCT/US2019/013991 WO2019143803A1 (en) | 2018-01-17 | 2019-01-17 | Adeno-associated virus gene therapy for 21-hydroxylase deficiency |
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| JP2023062403A Division JP2023076698A (en) | 2018-01-17 | 2023-04-06 | Adeno-associated virus gene therapy for 21-hydroxylase deficiency |
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| JPWO2019143803A5 true JPWO2019143803A5 (en) | 2022-01-26 |
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| JP2023062403A Pending JP2023076698A (en) | 2018-01-17 | 2023-04-06 | Adeno-associated virus gene therapy for 21-hydroxylase deficiency |
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| JP2023062403A Pending JP2023076698A (en) | 2018-01-17 | 2023-04-06 | Adeno-associated virus gene therapy for 21-hydroxylase deficiency |
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| EP (1) | EP3740244A1 (en) |
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| AR125143A1 (en) | 2021-03-19 | 2023-06-14 | Adrenas Therapeutics Inc | GENE THERAPIES FOR 21-HYDROXYLASE DEFICIENCY |
| CA3235145A1 (en) | 2021-10-12 | 2023-04-20 | Bridgebio Gene Therapy Llc | Methods and compositions for treating leukodystrophies |
| CN115976004A (en) * | 2022-12-27 | 2023-04-18 | 天津科技大学 | Progesterone 17 alpha-hydroxylase mutant and application thereof |
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| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| AU762220B2 (en) * | 1998-05-28 | 2003-06-19 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | AAV5 vector and uses thereof |
| EP2573170B1 (en) | 2001-12-17 | 2017-12-20 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor |
| PT1453547T (en) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| PT3211085T (en) | 2003-09-30 | 2021-06-17 | Univ Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| JP6091435B2 (en) | 2011-02-22 | 2017-03-08 | カリフォルニア インスティチュート オブ テクノロジー | Protein delivery using adeno-associated virus (AAV) vectors |
| WO2015013313A2 (en) | 2013-07-22 | 2015-01-29 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| WO2015041718A1 (en) * | 2013-09-20 | 2015-03-26 | The General Hospital Corporation | Uses of modified mullerian inhibiting substance (mis) proteins for the treatment of neurodegenerative diseases |
| EP3054006A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography |
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