Tag: gene therapy

  • Factor Ix Rec, Fc Fusion Protn Solution, Reconstituted (Recon Soln)

    Factor Ix Rec, Fc Fusion Protn Solution, Reconstituted (Recon Soln)

    Discover the transformative impact of Factor IX Rec, Fc Fusion Protein Solution in managing hemophilia B. This biologically engineered treatment not only increases Factor IX levels for effective blood clotting but also enhances patients’ quality of life, allowing them to engage in everyday activities with confidence. Learn about symptoms, diagnosis, treatment options, and crucial support…

  • Transthyretin Methionine-30 Amyloidosis (Type I)

    Transthyretin Methionine-30 Amyloidosis (Type I): Definition and Description of Transthyretin Methionine-30 Amyloidosis (Type I): Transthyretin Methionine-30 Amyloidosis (ATTR-M30), a hereditary form of amyloidosis, is characterized by the deposition of misfolded transthyretin (TTR) protein in tissues and organs. The disease arises from a mutation in the TTR gene on chromosome 18, specifically within the codon for…

  • Spinal Muscular Atrophy (SMA)

    Spinal Muscular Atrophy (SMA): Definition and Description of Spinal Muscular Atrophy (SMA): Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by the loss of motor neurons in the spinal cord, leading to muscle weakness and atrophy. It is caused by a deficiency of the survival motor neuron (SMN) protein, which is essential for the…

  • RP

    RP: Definition and Description of RP: ‘RP’, or Retinitis Pigmentosa, is a genetic eye disorder that leads to progressive degeneration of the retina. This condition affects the photoreceptor cells, leading to a gradual loss of vision. Medically, RP is characterized by the deterioration of rod and cone cells, which are crucial for night vision and…

  • Cloning

    Cloning Definition and Description of Cloning Cloning refers to the process of creating a genetically identical copy of an organism, cell, or DNA fragment. In the medical context, cloning can involve various types of cloning techniques such as somatic cell nuclear transfer (SCNT), molecular cloning, and reproductive cloning. The term has various implications in genetics,…

  • Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

    Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Definition and Description of Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) is a rare genetic disorder characterized by the absence or dysfunction of the enzyme adenosine deaminase, which is crucial for the proper development and functioning of the immune system. This condition is categorized…