公开(公告)号：US20140011977A1

公开(公告)日：2014-01-09

申请号：US13901476

申请日：2013-05-23

Applicant: Cold Spring Harbor Laboratory

Inventor： Adrian R. Krainer ,  Luca Cartegni

IPC: C12N15/113

CPC classification number: C12N15/1135 ,  A61K38/00 ,  A61K48/00 ,  C07K2319/00 ,  C12N15/113 ,  C12N2310/15 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2310/52 ,  C12N2310/3521

Abstract: The present invention provides a chimeric molecule including a base-pairing segment that binds specifically to a single-stranded nucleic acid molecule; and a moiety that modulates splicing or translation. The invention also provides a chimeric molecule including a base-pairing segment that binds specifically to a double-stranded nucleic acid molecule; and a peptide that modulates transcription, wherein the peptide comprises up to about one hundred amino acid residues.

Abstract translation: 本发明提供了包含与单链核酸分子特异性结合的碱基配对区段的嵌合分子; 以及调节拼接或翻译的部分。 本发明还提供了嵌合分子，其包括与双链核酸分子特异性结合的碱基配对区段; 和调节转录的肽，其中肽包含多至约一百个氨基酸残基。

公开(公告)号：US20130274704A1

公开(公告)日：2013-10-17

申请号：US13908057

申请日：2013-06-03

Applicant: Cold Spring Harbor Laboratory

Inventor： Timothy P. Tully ,  Filippo Cavalieri

IPC: A61N1/30

CPC classification number: A61K38/1709 ,  A61K31/00 ,  A61K48/00 ,  A61N1/30 ,  G09B19/00

Abstract: The present invention provides methods of therapy of cognitive deficits associated with a central nervous system disorder or condition, methods of enhancing cognitive performance and methods for repeated stimulation of neuronal activity or a pattern of neuronal activity, such as that underlying a specific neuronal circuit(s). The methods comprise combining cognitive training protocols and a general administration of CREB pathway-enhancing agents.

公开(公告)号：US20130109091A1

公开(公告)日：2013-05-02

申请号：US13720474

申请日：2012-12-19

Applicant: Isis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2320/33 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

公开(公告)号：US20030032577A1

公开(公告)日：2003-02-13

申请号：US09999779

申请日：2001-10-25

Applicant: Cold Spring Harbor Laboratory

Inventor： Grigori N. Enikolopov ,  Natalia I. Peunova ,  Boris A. Kuzin ,  Hollis Cline ,  Tatyana Michurina

IPC: A61K045/00 ,  A61K031/00

CPC classification number: A61K31/223 ,  A61K31/00 ,  A61K31/155 ,  A61K31/17 ,  A61K31/198 ,  A61K31/216

Abstract: The present invention is based on the discovery that nitric oxide (NO) is an important growth regulator in an intact developing organism. In particular, the present invention relates to a method of increasing in a mammal a population of hematopoietic stem cells which are capable of undergoing normal hematopoiesis, differentiation and maturation in hematopoietic tissue, wherein the hematopoietic tissue is contacted with multiple doses of at least one inhibitor of NO, such as one or more inhibitors of nitric oxide synthase (NOS), thereby producing hematopoietic tissue having an increased population of hematopoietic stem cells which are capable of undergoing normal hematopoiesis, differentiation and maturation. The present invention also relates to a method of increasing a population of cells in S phase in a tissue of a mammal, comprising contacting the tissue with multiple doses of at least one inhibitor (one or more) of NO, such as an inhibitor of NOS. The invention also pertains to a method of regenerating tissue in an adult mammal comprising contacting a selected tissue (e.g., blood, skin, bone and digestive epithelium), or precursor cells of the selected tissue, with multiple doses of at least one inhibitor (one or more) of NO, thereby inhibiting differentiation and inducing proliferation of cells of the tissue.

公开(公告)号：US20250028193A1

公开(公告)日：2025-01-23

申请号：US18906958

申请日：2024-10-04

Applicant: The Trustees of Columbia University in the City of New York ,  COLD SPRING HARBOR LABORATORY

Inventor： Michal Lipson ,  Aseema Mohanty ,  Mohammad Amin Tadayon ,  Adam Kepecs ,  Qian Li ,  Xingchen Ji ,  Christine P. Hendon ,  Xinwen Yao

IPC: G02F1/01 ,  A61N5/06

Abstract: Provided are devices that have a distal portion configured to be implanted in a brain of a subject. The distal portion includes one or more emitters configured to emit light in the visible spectrum. The device includes a proximal portion configured to be external to the brain of the subject while the distal portion is implanted, wherein the proximal portion includes at least one waveguide in optical communication with the one or more emitters. The at least one waveguide defines a cross-sectional width less than 500 nm. The at least one waveguide is optionally coupled to a heating element that is optionally configured to adjust a phase of light within the at least one waveguide.

公开(公告)号：US12178825B2

公开(公告)日：2024-12-31

申请号：US17866724

申请日：2022-07-18

Applicant: COLD SPRING HARBOR LABORATORY ,  DEPYMED, INC.

Inventor： Nicholas Tonks ,  Navasona Krishnan ,  Andreas Grill ,  Howard Sard

IPC: A61K31/58 ,  A61K31/4184

Abstract: Provided is a method of forming a copper-containing complex, including contacting a sample containing copper with a compound of Formula I: wherein R is —OH or —O—CH3. Also provided is a method of inhibiting enzymatic activity of a kinase in a sample, including contacting the sample with a compound of Formula I. Further provided is a method of administering to a subject a pharmaceutical composition including a compound of Formula I optionally complexed with copper. Also provided is a pharmaceutical composition including copper complexed with a compound of Formula I.

公开(公告)号：US20240199561A1

公开(公告)日：2024-06-20

申请号：US18261563

申请日：2022-01-19

Applicant: COLD SPRING HARBOR LABORATORY ,  THE FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH ,  VICHEM CHEMIE RESEARCH, LTD.

Inventor： Nicholas TONKS ,  Carla-Maria GAUSS ,  Prabhadevi VENKATARAMANI ,  Leemor JOSHUA-TOR ,  Elad ELKAYAM ,  Yousef Al ABED ,  Kai Fan CHENG ,  Ahmad ALTITI ,  Laszlo ORFI ,  Istvan SZABADKAI ,  Balint SZOKOL ,  Zoltan HORVATH

IPC: C07D277/66 ,  A61K31/428 ,  A61K31/431 ,  A61K31/506 ,  C07D513/04

CPC classification number: C07D277/66 ,  A61K31/428 ,  A61K31/431 ,  A61K31/506 ,  C07D513/04

Abstract: Provided is a compound, including a compound of Formula I:




or a pharmaceutically acceptable salt thereof, wherein W is a direct bond or an optionally substituted C, and if W is a direct bond, then X1, X2, X3, and X4 are each independently H, OH, or an electrophile, and optionally one pair selected from X1 and X2, X2 and X3, and X3 and X4 forms a five-membered ring including one or more heteroatom, wherein the one or more heteroatom is selected from O, N, and S, and the five-membered ring is optionally substituted with a ═C, an ═S, or an electrophile, and Y1, Y2, Y3, Y4 and Y5 are each independently selected from H, CH3, OH, O—CH3, and NO2, and an electrophile, and optionally Y2 and Y3 form a five-membered ring including one or more heteroatom, wherein the one or more heteroatom is selected from O, N, and S, and the five-membered ring is optionally substituted with a ═C, ═S, or an electrophile.

公开(公告)号：US20230302036A1

公开(公告)日：2023-09-28

申请号：US18005514

申请日：2021-07-13

Applicant: CONSEJO NACIONAL DE INVESTIGACIONES CIENTIFICAS Y TECNICAS ,  UNIVERSIDAD DE BUENOS AIRES ,  COLD SPRING HARBOR LABORATORY

Inventor： Luciano E. MARASCO ,  Alberto R. KORNBLIHTT ,  Adrian KRAINER ,  Jose STIGLIANO

IPC: A61K31/7125 ,  A61K31/711 ,  C12N15/113

CPC classification number: A61K31/7125 ,  A61K31/711 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/31

Abstract: The present disclosure relates compositions and methods to treat neuromuscular diseases and disorders, e.g., spinal muscular atrophy (SMA), characterized by the presence of a splicing silencer located in SMN2 intron 7 pre-mRNA, comprising coadministering a therapeutically effective amount of an antisense oligonucleotide (ASO) complementary to a nucleotide sequence within intron 7 of human SMN2 pre-mRNA; and a subclinical dose of a histone deacetylate inhibitor, e.g., valproic acid, trichostatin A, or a combination thereof.

公开(公告)号：US20230235348A1

公开(公告)日：2023-07-27

申请号：US17813577

申请日：2022-07-19

Applicant: Cold Spring Harbor Laboratory ,  Cambridge Enterprise Limited

Inventor： Robert Martienssen ,  Charles Underwood ,  Ian Henderson

IPC: C12N15/82 ,  A01H1/02 ,  C12N15/113

CPC classification number: C12N15/8261 ,  A01H1/02 ,  C12N15/113 ,  C12N15/8218 ,  Y02A40/146

Abstract: The present disclosure provides methods for increasing meiotic recombination in crop plants, as well as plants and seeds produced by such methods.

公开(公告)号：US20220370479A1

公开(公告)日：2022-11-24

申请号：US17866724

申请日：2022-07-18

Applicant: COLD SPRING HARBOR LABORATORY ,  DEPYMED, INC.

Inventor： Nicholas TONKS ,  Navasona KRISHNAN ,  Andreas GRILL ,  Howard SARD

IPC: A61K31/58 ,  A61K31/4184

Abstract: Provided is a method of forming a copper-containing complex, including contacting a sample containing copper with a compound of Formula I: wherein R is —OH or —O—CH3. Also provided is a method of inhibiting enzymatic activity of a kinase in a sample, including contacting the sample with a compound of Formula I. Further provided is a method of administering to a subject a pharmaceutical composition including a compound of Formula I optionally complexed with copper. Also provided is a pharmaceutical composition including copper complexed with a compound of Formula I.