C12N2310/344

NUCLEIC ACID MOLECULE HAVING IMPROVED STABILITY, AND USE THEREOF

Provided are structural properties of double-stranded RNA molecules for inducing RNA interference, and more specifically, a novel nucleic acid molecule structure for inducing RNAi with improved stability, and a use thereof. A nucleic acid molecule for inducing RNAi provided herein is capable of significantly improving stability of a double-stranded RNA molecule in vivo while maintaining inhibition efficiency for a target gene, and therefore, as a expandable platform for targeting various genes, the RNA nucleic acid molecule is expected to replace siRNA molecules in the art, and be usefully utilized in the field of diagnosis or treatment of various diseases in research and clinical fields.

Optimized anti-FLT1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders

This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.

TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, β-thalassemia (e.g., β-thalassemia major and β-thalassemia intermiedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.

Compositions and Methods for Treating Alpha-1 Antitrypsin Deficiency

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

Compositions and methods for treating alpha-1 antitrypsin deficiency

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

MAPT RNA INTERFERENCE AGENTS

Provided herein are MAPT RNAi agents and compositions comprising a MAPT RNAi agent. Also provided herein are methods of using the MAPT RNAi agents or compositions comprising a MAPT RNAi agent for reducing MAPT expression and/or treating tauopathy in a subject.

Modulators of FOXP3 expression

The present embodiments provide methods, compounds, and compositions useful for inhibiting FOXP3 expression, which may be useful for treating, preventing, or ameliorating cancer.

NUCLEIC ACID NANOPARTICLES, PHARMACEUTICAL COMPOSITION COMPRISING SAME, DRUG COMPRISING DOXORUBICIN AND PREPARATION METHOD THEREFOR
20220409742 · 2022-12-29 ·

Disclosed are nucleic acid nanoparticles, a pharmaceutical composition comprising the same, a drug comprising doxorubicin and a preparation method thereof. The nucleic acid nanoparticles have a nucleic acid structural domain, the nucleic acid structural domain includes a sequence a, a sequence b and a sequence c; the sequence a includes a sequence a1 or a sequence obtained by insertion, deletion or substitution of at least one base in the sequence a1, the sequence b includes a sequence b1 or a sequence obtained by insertion, deletion or substitution of at least one base in the sequence b1 and the sequence c includes a sequence d or a sequence obtained by insertion, deletion or substitution of at least one base in the sequence.

RANGE INFORMATION DETECTION USING COHERENT PULSE SETS WITH SELECTED WAVEFORM CHARACTERISTICS
20220413140 · 2022-12-29 ·

Method and apparatus for obtaining range information associated with a target using light detection and ranging (LiDAR). An emitter transmits a set of pulses of electromagnetic radiation to illuminate a target. The set of pulses includes a pair of emitted pulses with different waveform characteristics, such as slightly different phases. A detector receives a reflected set of pulses from the target. The received set of pulses includes a pair of received pulses with corresponding different waveform characteristics. The detector determines the range information by decoding the received pulses, such as by calculating an average of the phase differential in the received pulses. In this way, a single stage detector can be used without the need for separate I/Q (in-phase and quadrature) channels. Phase chirping can be used so that each successive pair of pulses has a different phase difference. Other waveform characteristics can be used including frequency, amplitude, shape, etc.

COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.