C12Y207/11017

METHODS AND COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
20240058475 · 2024-02-22 ·

Some embodiments of the methods and compositions provided herein relate to treating, inhibiting or ameliorating a skeletal muscular dystrophy with a polynucleotide encoding a SERCA2a polypeptide. In some embodiments, the muscular dystrophy comprises Duchenne muscular dystrophy (DMD) or Becker's muscular dystrophy (BMD). In some embodiments, the polynucleotide comprises a viral vector, such as an adeno-associated viral (AAV) vector. More embodiments include methods and compositions to screen for a therapeutic agent to treat, inhibit or ameliorate a skeletal muscular dystrophy in which the screen comprises an in vitro ventricular cardiac tissue model.

TISSUE RETRACTOR AND METHODS OF USE
20190321022 · 2019-10-24 ·

Systems, devices and methods related to retractor systems that retract tissue to allow access to a surgical site are described. The retractor systems can include a first rotatable arm attachable to a first blade, a second rotatable arm attachable to a second blade and a third linearly translatable arm attachable to a third blade. An attachment mechanism that is attachable to a fourth blade can be removably coupled to a mount on the frame. The retractors systems can also include two blade systems whereby when the retractor is in a closed configuration, the two blades form an oval opening.

CAMK2D ANTISENSE OLIGONUCLEOTIDES AND USES THEREOF

The present disclosure relates to antisense oligonucleotides, which target CAMK2D mRNA in a cell, leading to reduced expression of CAMK2D protein. Reduction of CAMK2D protein expression is beneficial for the treatment of certain medical disorders, e.g., cardiovascular-related diseases or disorders.

CaMKK-β as a target for treating cancer

Provided herein are compounds, compositions, including pharmaceutical compositions, having anti-cancer activity. Also provided are methods for diagnosing, detecting, and treating cancer in a subject, as well as a method for evaluating cancer stage in a subject, wherein the methods include determining the amount of a Ca.sup.2+/calmodulin dependent kinase kinase (CaMKK) in a sample. Further provided are methods of screening and identifying a compound that inhibits CaMKK.

EXTRA-HEPATIC DELIVERY IRNA COMPOSITIONS AND METHODS OF USE THEREOF

The present invention provides double stranded ribonucleic acid (dsRNA) agents for inhibiting expression of a target gene, comprising an antisense strand which is complementary to the target gene; a sense strand which is complementary to the antisense strand and forms a double stranded region with the antisense strand; and one or more C.sub.22 hydrocarbon chains conjugated to one or more internal positions on at least one strand, compositions comprising such dsRNA agents, and methods of use thereof for treating a subject having a skeletal muscle disorder, a cardiac muscle disorder, or an adipose tissue disorder.

METHOD FOR FORMING A REVERSIBLE PROTEIN NANOCLUSTER USING LIGHT IN A CELL
20170275346 · 2017-09-28 ·

To efficiently analyze interaction and function between proteins, the present invention relates to a method for forming a light-induced protein nanocluster, comprising: an expression vector preparation step of preparing a first expression vector including polynucleotides coding a first fusion protein including a light-induced heterodimer-forming protein and a first self-assembly protein, and a second expression vector including polynucleotides coding a couple protein that forms a homodimer with said light-induced heterodimer-forming protein, or a second fusion protein including said couple protein and a second self-assembly protein; a transformed cell, tissue or individual preparation step of transforming cells, tissues or individuals using said first expression vector and second expression vector; and a light radiation step of radiating light having a wavelength for inducing the formation of heterodimer between said light-induced heterodimer-forming protein and said couple protein, to said transformed cells, tissue or individuals.

Method for forming a reversible protein nanocluster using light in a cell

To efficiently analyze interaction and function between proteins, the present invention relates to a method for forming a light-induced protein nanocluster, comprising: an expression vector preparation step of preparing a first expression vector including polynucleotides coding a first fusion protein including a light-induced heterodimer-forming protein and a first self-assembly protein, and a second expression vector including polynucleotides coding a couple protein that forms a homodimer with said light-induced heterodimer-forming protein, or a second fusion protein including said couple protein and a second self-assembly protein; a transformed cell, tissue or individual preparation step of transforming cells, tissues or individuals using said first expression vector and second expression vector; and a light radiation step of radiating light having a wavelength for inducing the formation of heterodimer between said light-induced heterodimer-forming protein and said couple protein, to said transformed cells, tissue or individuals.

CALCIUM REPORTER GENE

As a calcium indicator protein having an excellent fluorescent characteristic and calcium reactivity, there is provided DNA in which one of a nucleotide sequence derivative of a calmodulin-binding sequence (ckkap sequence) of calcium/calmodulin-dependent protein kinase kinase and a nucleotide sequence encoding a calcium-binding sequence (CaM sequence) of calmodulin is linked to a 5 end of a nucleotide sequence encoding a fluorescent protein, and the other nucleotide sequence is linked to a 3 end of the nucleotide sequence encoding the fluorescent protein. The calcium indicator protein encoded by this DNA, which based on the derivative of the ckkap sequence as a binding domain for the calcium-bound CaM sequence, exhibits a fluorescent characteristic and calcium reactivity superior to those of conventional calcium indicator proteins.

COMPOSITIONS AND METHODS FOR TREATING A CARDIAC DISEASE

The invention of the disclosure features Ca.sup.2+-calmodulin dependent kinase II (CaMKII) inhibitory multimeric polypeptides polynucleotides encoding such polypeptide, and methods of using the same for the treatment of cardiac diseases (e.g., catecholaminergic polymorphic ventricular tachycardia (CPVT) or atrial fibrillation (AF)).

CAMKK-BETA AS A TARGET FOR TREATING CANCER
20170027928 · 2017-02-02 ·

Provided herein are compounds, compositions, including pharmaceutical compositions, having anti-cancer activity. Also provided are methods for diagnosing, detecting, and treating cancer in a subject, as well as a method for evaluating cancer stage in a subject, wherein the methods include determining the amount of a Ca.sup.2+/calmodulin dependent kinase kinase (CaMKK) in a sample. Further provided are methods of screening and identifying a compound that inhibits CaMKK.