Patent classifications
A61K38/1709
CELLULAR REPROGRAMMING TO REVERSE AGING AND PROMOTE ORGAN AND TISSUE REGENERATION
Provided herein are engineered nucleic acids (e.g., expression vectors, including viral vectors, such as lentiviral vectors, adenoviral vectors, AAV vectors, herpes viral vectors, and retroviral vectors) that encode OCT4; KLF4; SOX2; or any combination thereof that are useful, for example, in inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging, or any combination thereof. Also provided herein are recombinant viruses (e.g., lentiviruses, alphaviruses, vaccinia viruses, adenoviruses, herpes viruses, retroviruses, or AAVs) comprising the engineered nucleic acids (e.g., engineered nucleic acids), engineered cells, compositions comprising the engineered nucleic acids, the recombinant viruses, engineered cells, engineered proteins, chemical agents that are capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, an engineered protein selected from the group consisting of OCT4; KLF4; SOX2; or any combination thereof, an antibody capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, and methods of treating a (e.g., ocular disease), preventing a disease (e.g., ocular disease), regulating (e.g., inducing or inducing and then stopping) cellular reprogramming, regulating tissue repair, regulating tissue regeneration, or any combination thereof).
CELLULAR REPROGRAMMING TO REVERSE AGING AND PROMOTE ORGAN AND TISSUE REGENERATION
Provided herein are engineered nucleic acids (e.g., expression vectors, including viral vectors, such as lentiviral vectors, adenoviral vectors, AAV vectors, herpes viral vectors, and retroviral vectors) that encode OCT4; KLF4; SOX2; or any combination thereof that are useful, for example, in inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging, or any combination thereof. Also provided herein are recombinant viruses (e.g., lentiviruses, alphaviruses, vaccinia viruses, adenoviruses, herpes viruses, retroviruses, or AAVs) comprising the engineered nucleic acids (e.g., engineered nucleic acids), engineered cells, compositions comprising the engineered nucleic acids, the recombinant viruses, engineered cells, engineered proteins, chemical agents that are capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, an engineered protein selected from the group consisting of OCT4; KLF4; SOX2; or any combination thereof, an antibody capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, and methods of treating a (e.g., ocular disease), preventing a disease (e.g., ocular disease), regulating (e.g., inducing or inducing and then stopping) cellular reprogramming, regulating tissue repair, regulating tissue regeneration, or any combination thereof).
IN VITRO AND IN VIVO GENE DELIVERY TO IMMUNE EFFECTOR CELLS USING NANOPARTICLES FUNCTIONALIZED WITH DESIGNED ANKYRIN REPEAT PROTEINS (DARPINS)
The present disclosure generally relates to therapies involving immune effector cells such as T cells engineered to express antigen receptors such as T cell receptors (TCRs) or chimeric antigen receptors (CARs). It is demonstrated herein that such antigen receptor-engineered immune effector cells may be generated in vitro/ex vivo as well as in vitro by delivering nucleic acid encoding an antigen receptor for genetic modification to cells using particles comprising the nucleic acid and a targeting molecule for targeting the immune effector cells, wherein the targeting molecule is a designed ankyrin repeat protein (DARPin). In particular, DARPins are described herein which are high-affinity binders for CDS binding to the CDS receptor on human and non-human primate (NHP) cells. Nanoparticles functionalized with CD8− targeting DARPins (CDS-DARPin) can deliver genes exclusively and specifically to human CD8.sup.+ T cells in vitro and in vivo.
METHOD FOR PRODUCING STEM CELL WITH ENHANCED EFFICACY
Disclosed is a method for producing a stem cell with enhanced efficacy, a stem cell produced thereby, and a method for treating a disease using the same.
According to the present invention, sizes of stem cells may be reduced and proliferation ability, differentiation potency, migration ability, and angiogenic potential of stem cells may be significantly enhanced by applying a culturing method provided herein. Thus the present invention may be usefully applied to development of therapeutic agents using stem cells.
COMPOUNDS AND METHODS FOR TREATING OR REDUCING PRURITUS
The present invention features methods and compositions for preventing, inhibiting, or reducing the sensation of itch (pruritus) by modulating the gene expression of TMEM184B. Modulating the gene expression of TMEM184B may comprise increasing Tmem184B protein expression, increasing Tmem184B protein activity or both. Furthermore, the methods and compositions described herein may activate the Tmem184B protein or activate a pathway leading to Tmem184B activation. Additionally, the methods and compositions for modulating the gene expression of TMEM184B may be used to help prevent or treat certain diseases or conditions such as dermatitis.
Methods and compositions for treatment of multi-drug resistant tumors
Methods for treatment of a multi-drug resistant (MDR) tumor in a subject are disclosed. The methods comprise administering to the subject in need of treatment a therapeutically effective amount of an anti-mycoplasma agent and/or an agent blocking the interaction between membrane protein P37 of mycoplasma and Annexin A2 of host cells of the subject, prior to, at the same time with, or after chemotherapy. Relevant pharmaceutical compositions, kits, uses are also disclosed.
METHOD OF PROGNOSIS OF BRONCHOPULMONARY DYSPLASIA IN PREMATURE INFANTS
The present invention relates to a method for prognosis the risk of bronchopulmonary dysplasia in premature infant by adjusting a score obtained from lung ultrasound with the gestational age of the infant.
Methods and Compositions for Modulationg Heterochromatin Dysfunction, Genomic Instability, and Associate Conditions
The present invention includes a method of increasing, stimulating, inducing, promoting, enhancing or maintaining the genomic stability of a cell of a subject, the method comprising decreasing, reducing, inhibiting, suppressing, limiting or controlling loss of methylation of heterochromatin in the cell and/or modulating heterochromatin dysfunction in a cell of a subject, the method comprising activating, eliciting, stimulate ng, inducing, promoting, increasing or enhancing expression or activity in the cell of one or more DNA methyltransferase (DNMT).
ANTI-TUMOR POLYPEPTIDE Bax-BH3, FLUORESCENT POLYMERIC NANOMICELLE, PREPERATION METHOD AND USE THEREOF
The present disclosure provides an anti-tumor polypeptide Bax-BH3, a fluorescent polymeric nanomicelle, a preparation method and use thereof, belonging to the technical field of medicines. The anti-tumor polypeptide Bax-BH3 has an amino acid sequence set forth in SEQ ID No: 1; the fluorescent polymeric nanomicelle includes the anti-tumor polypeptide Bax-BH3 and a polymer carrier; and the polymer carrier is a block copolymer RGD-PHPMA-b-Poly(MMA-alt-(Rhob-MA)). In the present disclosure, the anti-tumor polypeptide Bax-BH3 has desirable biocompatibility and biological activity; and the fluorescent polymeric nanomicelle encapsulates the anti-tumor polypeptide Bax-BH3 by the block copolymer RGD-PHPMA-b-Poly(MMA-alt-(Rhob-MA)), with high encapsulation rate and drug loading, and good release performance.
SMAC/Diablo inhibitors useful for treating cancer
The present invention relates to compositions and methods for treating cancer, particularly to agents that inhibit the expression and/or activity of the protein second mitochondria-derived activator of caspase/direct inhibitor of apoptosis-binding protein with low pI (SMAC/Diablo). The inhibiting agents include RNA interference molecules silencing the expression of SMAC/Diablo and peptides modulating its interactions within the cell nucleus and mitochondria. The methods and agents of the present invention are useful in treating cancers associated with overexpression of SMAC/Diablo.