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  2. Branched chain amino acid transaminase 1 - Wikipedia

    en.wikipedia.org/wiki/Branched_chain_amino_acid...

    Branched chain amino acid transaminase 1 is a protein that in humans is encoded by the BCAT1 gene. [5] It is the first enzyme in the branched-chain amino acid (BCAA) degradation pathway and facilitates the reversible transamination of BCAAs and glutamate. BCAT1 resides in the cytoplasm, while its isoform, BCAT2, is found in the mitochondria.

  3. Branched-chain amino acid aminotransferase - Wikipedia

    en.wikipedia.org/wiki/Branched-chain_amino_acid...

    BCAT1 is located in the mitochondria, BCAT2, 3, and 5 are located in chloroplasts, and BCAT4 and 6 are located in the cytoplasm of A. thaliana. [23] However, studies of BCATs in Solanum tuberosum (potato) revealed two isoforms that are 683 (BCAT1) and 746 (BCAT2) bp long located primarily in chloroplasts. [24]

  4. VEXAS syndrome - Wikipedia

    en.wikipedia.org/wiki/VEXAS_syndrome

    In 2022 the National Cancer Institute announced a three-year clinical trial to evaluate stem cell transplant as a possible treatment for patients with VEXAS. [13] Scientists, including David B. Beck, one of the original discoverers, at the New York University Grossman School of Medicine and NYU Langone Health were also actively researching the ...

  5. FDA approves Pfizer's first gene therapy for rare inherited ...

    www.aol.com/news/fda-approves-pfizer-first-gene...

    The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...

  6. NEMO deficiency syndrome - Wikipedia

    en.wikipedia.org/wiki/NEMO_deficiency_syndrome

    It is the mainstay of treatment for patients affected by primary antibody deficiency. In addition to immunoglobulin treatment, children may need to take antibiotics or antifungal medicines to prevent infections or treat them promptly when they occur. Regular monitoring and check-ups will help to catch infections early.

  7. Is gene therapy the next big step in vision loss treatment? - AOL

    www.aol.com/gene-therapy-next-big-step-120000520...

    Mutations in more than 300 genes characterized so far are known to play a causal role in inherited retinal diseases. Gene therapy approaches offer hope for the treatment of these diseases that ...

  8. FDA considers first CRISPR gene editing treatment that may ...

    www.aol.com/fda-considers-first-crispr-gene...

    The FDA said treatment for severe sickle cell is an “unmet medical need.” When someone has sickle cell disease their red blood cells don’t function the way they should. Red blood cells are ...

  9. Hereditary neuropathy with liability to pressure palsy

    en.wikipedia.org/wiki/Hereditary_neuropathy_with...

    PMP22 point mutations, such as the frameshift mutation Gly94fsX222 (c.281_282insG), can cause clinical overlap between PNPP and Charcot–Marie–Tooth disease type 1A. Missense, nonsense, and splice site mutations have been described. [10] PMP22 encodes a 22-kD protein that comprises 2 to 5% of peripheral nervous system myelin. [11]