A61K38/42

SILK PARTICLES ENCAPSULATING OXYGEN CARRIERS AS ARTIFICIAL BLOOD SUBSTITUTE
20230321200 · 2023-10-12 ·

Described are silk fibroin nanoparticles encapsulating oxygen carriers and compositions containing silk fibroin nanoparticles encapsulating oxygen carriers. Also described are methods of using the silk fibroin nanoparticles encapsulating oxygen carriers as artificial blood substitutes.

METHODS AND COMPOSITIONS FOR TRANSDUCING HEMATOPOIETIC STEM AND PROGENITOR CELLS IN VIVO

The invention relates to the in vivo transduction of hematopoietic stem and progenitor cells (HSPCs) in a subject, such as a human subject, and to the treatment of subjects suffering from various pathologies, such as blood diseases, metabolic disorders, cancers, and autoimmune diseases, among others.

METHODS AND COMPOSITIONS FOR TRANSDUCING HEMATOPOIETIC STEM AND PROGENITOR CELLS IN VIVO

The invention relates to the in vivo transduction of hematopoietic stem and progenitor cells (HSPCs) in a subject, such as a human subject, and to the treatment of subjects suffering from various pathologies, such as blood diseases, metabolic disorders, cancers, and autoimmune diseases, among others.

METHOD FOR ACTIVATING EXPRESSION OF GAMMA-GLOBIN GENE, AND COMPOSITION
20230310506 · 2023-10-05 ·

Provided is a new method for activating transcription of a gamma-globin gene. The method uses a single-stranded oligonucleotide (ssODN) containing GATA or an antisense complementary sequence TATC thereof as guidance information, and performs gene editing in a gamma-globin gene promoter region to form a GATA-containing enhancer element, which can promote the expression of the gamma-globin gene in mature red blood cells. Hematopoietic stem cells genetically edited by the method have normal functions, can significantly improve the expression of fetal hemoglobin after being differentiated into red blood cells, and therefore can be used in clinical treatment of beta-thalassemia and sickle cell anemia.

METHOD FOR ACTIVATING EXPRESSION OF GAMMA-GLOBIN GENE, AND COMPOSITION
20230310506 · 2023-10-05 ·

Provided is a new method for activating transcription of a gamma-globin gene. The method uses a single-stranded oligonucleotide (ssODN) containing GATA or an antisense complementary sequence TATC thereof as guidance information, and performs gene editing in a gamma-globin gene promoter region to form a GATA-containing enhancer element, which can promote the expression of the gamma-globin gene in mature red blood cells. Hematopoietic stem cells genetically edited by the method have normal functions, can significantly improve the expression of fetal hemoglobin after being differentiated into red blood cells, and therefore can be used in clinical treatment of beta-thalassemia and sickle cell anemia.

MEDICINE CONTAINING METHEMOGLOBIN ENDOPLASMIC RETICULUM AS ACTIVE INGREDIENT AND USE THEREOF

The present invention is intended to provide a medicament for cyanide poisoning, hydrogen sulfide poisoning, and azide poisoning, wherein the medicament does not cause side effects, is excellent in immediate effectiveness, and has a high antidotal effect. Specifically, the present invention relates to a medicament for cyanide poisoning, hydrogen sulfide poisoning, and azide poisoning, wherein the medicament comprises, as an active ingredient(s), one or more selected from the group consisting of a heme protein capable of binding to cyanide ions (CN.sup.−), hydrogen sulfide ions (HS.sup.−) and azide ions (N.sub.3.sup.−), a substance containing a heme protein capable of binding to CN.sup.−, HS.sup.− and N.sub.3.sup.−, and a heme derivative capable of binding to CN.sup.−, HS.sup.− and N.sub.3.sup.−. More specifically, the present invention relates to a medicament or a pharmaceutical composition for the treatment or prophylaxis of cyanide poisoning, hydrogen sulfide poisoning, and azide poisoning.

MEDICINE CONTAINING METHEMOGLOBIN ENDOPLASMIC RETICULUM AS ACTIVE INGREDIENT AND USE THEREOF

The present invention is intended to provide a medicament for cyanide poisoning, hydrogen sulfide poisoning, and azide poisoning, wherein the medicament does not cause side effects, is excellent in immediate effectiveness, and has a high antidotal effect. Specifically, the present invention relates to a medicament for cyanide poisoning, hydrogen sulfide poisoning, and azide poisoning, wherein the medicament comprises, as an active ingredient(s), one or more selected from the group consisting of a heme protein capable of binding to cyanide ions (CN.sup.−), hydrogen sulfide ions (HS.sup.−) and azide ions (N.sub.3.sup.−), a substance containing a heme protein capable of binding to CN.sup.−, HS.sup.− and N.sub.3.sup.−, and a heme derivative capable of binding to CN.sup.−, HS.sup.− and N.sub.3.sup.−. More specifically, the present invention relates to a medicament or a pharmaceutical composition for the treatment or prophylaxis of cyanide poisoning, hydrogen sulfide poisoning, and azide poisoning.

COMPOSITION FOR UPPER RESPIRATORY TRACT ADMINISTRATION AND METHOD THEREOF

The present invention discloses a composition for upper respiratory tract administration comprising a cyanide antidote and a metal chelator, and the composition is used for cure or protection of fire injury. When carried by a proper carrier, the composition is administrated to a subject in need via forms including a spray, an inhaler, or drops so as to prevent or cure injuries caused by hazardous substances in a fireground.

COMPOSITION FOR UPPER RESPIRATORY TRACT ADMINISTRATION AND METHOD THEREOF

The present invention discloses a composition for upper respiratory tract administration comprising a cyanide antidote and a metal chelator, and the composition is used for cure or protection of fire injury. When carried by a proper carrier, the composition is administrated to a subject in need via forms including a spray, an inhaler, or drops so as to prevent or cure injuries caused by hazardous substances in a fireground.

CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR

Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.