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Functionally-modified oligonucleotides and subunits thereof

HANSON GUNNAR J / WELLER DWIGHT D / CAI BAO ZHONG / ZHOU MING

H: sxd\lnterwoven\NRPortbl\DCC\SXD\11846421_1.docx-28/10/2016 Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5'-end groups are provided. The disclosed 5 compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects. N HCO2Et H0 N O S Ok N O Ci 0 N 1. X-PO N 2. 4-cyanopyridine/TFA H o 3. DIPEA, DCM, IPA O 0Base NHCO2Et Ity trityl o N 0 O) O 1. 0 CN) X{ O 2. 4-cyanopyridine/TFA 3. DIPEA, DCM, IPA XPO 0 Base I Base trityl N repeat cycle H

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Functionally-modified oligonucleotides and subunits thereof

HANSON GUNNAR J / WELLER DWIGHT D / CAI BAO ZHONG / ZHOU MING

H: sxd\lnterwoven\NRPortbl\DCC\SXD\11846421_1.docx-28/10/2016 Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5'-end groups are provided. The disclosed 5 compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects. N HCO2Et H0 N O S Ok N O Ci 0 N 1. X-PO N 2. 4-cyanopyridine/TFA H o 3. DIPEA, DCM, IPA O 0Base NHCO2Et Ity trityl o N 0 O) O 1. 0 CN) X{ O 2. 4-cyanopyridine/TFA 3. DIPEA, DCM, IPA XPO 0 Base I Base trityl N repeat cycle H

Functionally-modified oligonucleotides and subunits thereof

HANSON GUNNAR J / WELLER DWIGHT D / CAI BAO ZHONG / ZHOU MING

H: sxd\lnterwoven\NRPortbl\DCC\SXD\11846421_1.docx-28/10/2016 Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5'-end groups are provided. The disclosed 5 compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects. N HCO2Et H0 N O S Ok N O Ci 0 N 1. X-PO N 2. 4-cyanopyridine/TFA H o 3. DIPEA, DCM, IPA O 0Base NHCO2Et Ity trityl o N 0 O) O 1. 0 CN) X{ O 2. 4-cyanopyridine/TFA 3. DIPEA, DCM, IPA XPO 0 Base I Base trityl N repeat cycle H

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Title:

Functionally-modified oligonucleotides and subunits thereof

Contributors:

HANSON GUNNAR J (author) / WELLER DWIGHT D (author) / CAI BAO ZHONG (author) / ZHOU MING (author)

Publication date:

2018-11-01

Type of media:

Patent

Type of material:

Electronic Resource

Language:

English

Classification:

IPC: C04B Kalk , LIME

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