Modulation of gene expression

Tyler Chapman; Natalie Grima; Stephanie Rayner; Andrew Smith; Roger Chung; Kelly Williams; Marco Morsch

Modulation of gene expression

Tyler Chapman (Inventor), Natalie Grima (Inventor), Stephanie Rayner (Inventor), Andrew Smith (Inventor), Roger Chung (Inventor), Kelly Williams (Inventor), Marco Morsch (Inventor)

Research output: Patent

Abstract

The present disclosure relates generally to methods for modulating the expression of a heterologous transgene administered to a cell, and to nucleic acid constructs useful in such methods. The present disclosure also relates to the use of the nucleic acid constructs for enhancing motor neuron survival, inhibiting motor neuron degeneration and treating neurodegenerative conditions.

Original language	English
Patent number	WO2025007194
IPC	C12N 15/85 2006.1,A61K 48/00 2006.1,A61P 25/28 2006.1,C12Q 1/6897 2018.1,G01N 33/68 2006.1
Priority date	5/07/23
Publication status	Submitted - 9 Jan 2025

Bibliographical note

Designated States:
AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CV, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IQ, IR, IS, IT, JM, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, MG, MK, MN, MU, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, WS, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, CV, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SC, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, ME, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)

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title = "Modulation of gene expression",

abstract = "The present disclosure relates generally to methods for modulating the expression of a heterologous transgene administered to a cell, and to nucleic acid constructs useful in such methods. The present disclosure also relates to the use of the nucleic acid constructs for enhancing motor neuron survival, inhibiting motor neuron degeneration and treating neurodegenerative conditions.",

author = "Tyler Chapman and Natalie Grima and Stephanie Rayner and Andrew Smith and Roger Chung and Kelly Williams and Marco Morsch",

note = "Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CV, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IQ, IR, IS, IT, JM, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, MG, MK, MN, MU, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, WS, ZA, ZM, ZW African Regional Intellectual Property Organization (ARIPO) (BW, CV, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SC, SD, SL, ST, SZ, TZ, UG, ZM, ZW) Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM) European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, ME, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR) African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG); WO2025007194; C12N 15/85 2006.1,A61K 48/00 2006.1,A61P 25/28 2006.1,C12Q 1/6897 2018.1,G01N 33/68 2006.1",

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T1 - Modulation of gene expression

AU - Chapman, Tyler

AU - Grima, Natalie

AU - Rayner, Stephanie

AU - Smith, Andrew

AU - Chung, Roger

AU - Williams, Kelly

AU - Morsch, Marco

N1 - Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CV, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IQ, IR, IS, IT, JM, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, MG, MK, MN, MU, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, WS, ZA, ZM, ZW African Regional Intellectual Property Organization (ARIPO) (BW, CV, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SC, SD, SL, ST, SZ, TZ, UG, ZM, ZW) Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM) European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, ME, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR) African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)

PY - 2025/1/9

Y1 - 2025/1/9

N2 - The present disclosure relates generally to methods for modulating the expression of a heterologous transgene administered to a cell, and to nucleic acid constructs useful in such methods. The present disclosure also relates to the use of the nucleic acid constructs for enhancing motor neuron survival, inhibiting motor neuron degeneration and treating neurodegenerative conditions.

AB - The present disclosure relates generally to methods for modulating the expression of a heterologous transgene administered to a cell, and to nucleic acid constructs useful in such methods. The present disclosure also relates to the use of the nucleic acid constructs for enhancing motor neuron survival, inhibiting motor neuron degeneration and treating neurodegenerative conditions.

M3 - Patent

M1 - WO2025007194

ER -

Modulation of gene expression

Abstract

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