C12N9/0075

CYCLIC PEPTIDE INHIBITORS OF PSD-95 AND USES THEREOF

The present invention relates novel cyclic peptides which can act as inhibitors of protein-protein interactions, specifically by inhibiting the PDZ2 domain of PSD-95, as well as their use in treatment of excitotoxic-related diseases and neuropathic pain.

Modified bacteria for production of nitroaromatics

The disclosure relates, in some aspects, to compositions and methods useful for production of nitrated aromatic molecules. The disclosure is based, in part, on whole cell systems expressing artificial fusion proteins comprising cytochrome P450 enzymes linked to reductase enzymes. In some aspects, the disclosure relates to methods of producing nitrated aromatic molecules in whole cell systems having artificial fusion proteins comprising cytochrome P450 enzymes linked to reductase enzymes.

PLASMIN-RESISTANT PEPTIDES FOR TREATING STROKE AND RELATED CONDITIONS
20230055441 · 2023-02-23 ·

The invention provides variants of a previously described active agent for treating stroke, Tat-NR2B9c, in which target binding characteristics are retained by inclusion of L-amino acids at the C-terminus and plasmin-resistance is conferred by inclusion of D-amino acids elsewhere. An exemplary agent has the sequence ygrkkrrqrrrklssIETDV (SEQ ID NO:62). The resulting active agents have several advantages including administration at the same time as thrombolytic agents without significant loss of activity due to plasmin digestion. The resulting agents are also more suitable for administration by alternative routes to intravenous infusion, such as subcutaneous, intranasal and intramuscular, and for multi-dosing regimes for treatment of chronic conditions.

PERSISTENT MEMORY T-CELL RESPONSES TO CANCER AND INFECTIOUS-DISEASE ANTIGENS BY MANIPULATION OF AMINO ACID-CATABOLISM PATHWAYS

Provided herein are recombinant polynucleotides including a first nucleic acid sequence encoding an antigen, and a second nucleic acid sequence encoding an enzyme of an amino acid catabolic pathway. The provided recombinant polynucleotides are particularly useful for inducing long-lived immune responses having improved memory characteristics. Also provided are pharmaceutical compositions, viral particles, and host cells including the disclosed recombinant polynucleotides, and methods for using the disclosed materials.

2-aminoquinoline-based compounds for potent and selective neuronal nitric oxide synthase inhibition

Various 2-aminoquinoline compounds as can be used, in vivo or in vitro, for selective inhibition of neuronal nitric oxide synthase.

RNA preparations comprising purified modified RNA for reprogramming cells

The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and/or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and/or single-stranded run-on mRNAs.

COMPOUND FOR MODULATING DDAH AND ADMA LEVELS, AS WELL AS METHODS OF USING THEREOF TO TREAT DISEASE
20220144795 · 2022-05-12 ·

Disclosed are compounds that can modulate DDAH and the amount of asymmetric dimethylarginine (ADMA) in a subject. Also provided are pharmaceutical compositions comprising these compounds, as well as methods of using these compositions to treat and/or prevent diseases associated with elevated or low levels of DDAH and ADMA.

GENE THERAPY DNA VECTOR BASED ON GENE THERAPY DNA VECTOR VTVAF17
20220119837 · 2022-04-21 ·

Gene-therapeutic DNA vectors based on the VTvaf17 gene-therapeutic DNA vector have been created for the treatment of diseases characterized by impaired mucociliary transport and mucolytic function and the development of mucostasis, including cystic fibrosis. The gene therapy DNA vector contains the coding region of the CFTR therapeutic gene, or the NOS1 therapeutic gene, or the AQ1 therapeutic gene, or the AQ3 therapeutic gene, or the AQ5 therapeutic gene. Methods for their preparation or use are proposed, as well as strains for the production of a gene therapy vector.

IN VITRO METHOD FOR IDENTIFYING THORACIC AORTIC ANEURYSMS (TAA) IN A SUBJECT

The present invention refers to an In vitro method for screening for subjects at risk of developing thoracic aortic aneurysm (TAA) or a disease causing TAA comprising: (a) measuring the expression pattern or level of at least A Disintegrin And Metalloproteinase with Thrombospondin Motifs 1 (ADAMTS1) obtained from an isolated biological sample of the subjects to be screened; and (b) comparing said expression pattern or level of at least ADAMTS1 of the subjects to be screened with an already established expression pattern or level, wherein reduced expression of at least ADAMTS1 is indicative of a thoracic aortic aneurysm (TAA).

MODIFIED BACTERIA FOR PRODUCTION OF NITROAROMATICS
20230287466 · 2023-09-14 · ·

The disclosure relates, in some aspects, to compositions and methods useful for production of nitrated aromatic molecules. The disclosure is based, in part, on whole cell systems expressing artificial fusion proteins comprising cytochrome P450 enzymes linked to reductase enzymes. In some aspects, the disclosure relates to methods of producing nitrated aromatic molecules in whole cell systems having artificial fusion proteins comprising cytochrome P450 enzymes linked to reductase enzymes.