8 results about "Beta globin" patented technology

The invention belongs to the field of biological detection, and particularly relates to a blood protein characteristic polypeptide, a detection kit and application of the blood protein characteristic polypeptide to identification of adulteration and weight gain of hirudo nipponia. And the sequence of the blood protein marker polypeptide is LLGNVIVVVLAR. On the basis of constructing a species marker polypeptide identification method, a heterologous blood protein traceability analysis strategy is introduced, beta globin in blood of common livestock such as pigs, cattle, sheep, horses, donkeys and the like is subjected to multiple sequence alignment, and highly conservative species-independent marker polypeptide is screened out as a chemical marker; and a multi-reaction monitoring (MRM) non-standard quantitative peak area ratio judgment and detection method based on a liquid chromatography-triple quadrupole mass spectrometry technology is established, so that a scientific judgment basis is provided for the problem of blood doping and weight increment.

Provided are materials and methods for treating patients with hemoglobinopathies, either ex vivo or in vivo. Also provided are materials and methods for deleting and / or mutating a portion of a human beta globin locus on chromosome 11 and one or more of: a BCL11 A gene on chromosome 2, and a DNA sequence that encodes a transcriptional control region of the BCL11 A gene on chromosome 2, in a human cell by genome editing and thereby increasing the production of fetal hemoglobin (HbF) in the genome-edited human cells.

326 Abstract of the Disclosure The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin 5 polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency. 326 20 26 20 47 29 18 J un 2 02 6 1 8 J u n 2 0 2 6 2 0 2 6 2 0 4 7 2 9 3 2 6

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Disclosed herein are gene therapy vectors for efficient transduction of cells to express human beta globin genes. Specifically disclosed is an expression vector comprising: an expression cassette of a beta globin gene comprising an exon and an intron of a human beta globin gene, and a cis-acting element comprising one or more of WPRE, an SV40 polyadenylation signal and / or SV40 ori. The disclosed expression vectors have significantly enhanced viral vector packaging efficiency in viral vector packaging cell lines, which results in efficient integration of lentiviral vectors in target cells and high expression levels of beta globin genes. Pharmaceutical compositions and methods of treatment utilizing such expression vectors are also disclosed.

The invention provides anti-βS globin antibodies or antigen binding fragments thereof.

Materials and methods for treating a patient with hemoglobinopathy, both ex vivo and in vivo and materials and methods for deleting at least a portion of a human beta globin locus on chromosome 11 in a human cell by genome editing and thereby increasing the production of fetal hemoglobin (HbF).