Lentiviral vectors (LVs), derived from human immunodeficiency virus, are powerful tools for
modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells …

Researchers are looking into techniques to intervene sooner and earlier in the disease
process thanks to advances in disease genetics, etiologies, and prenatal diagnosis. We …

Monoclonal antibodies (mAbs) are commonly used biologic therapeutics with a wide variety
of clinical applications. During the development process, manufacturers consider different …

In recent decades, the use of adult multipotent stem cells has paved the way for the
identification of new therapeutic approaches for the treatment of monogenic diseases such …

Hemophilia A is the most common X-linked bleeding disorder affecting more than half-a-
million individuals worldwide. Persons with severe hemophilia A have coagulation FVIII …

Patients with the severe form of hemophilia A (HA) present with a severe phenotype, and
can suffer from life-threatening, spontaneous hemorrhaging. While prophylactic FVIII …

Introduction Placenta-derived mesenchymal cells (PLCs) endogenously produce FVIII,
which makes them ideally suited for cell-based fVIII gene delivery. We have previously …

Introduction Hemophilia A (HA) is the most common X-linked bleeding disorder, occurring in
1 in 5,000 live male births and affecting> 1 million individuals worldwide. Although advances …

Microfluidic technology enables recapitulation of organ-level physiology to answer pertinent
questions regarding biological systems that otherwise would remain unanswered. We have …

Medical literature has documented thousands of rare diseases, classified as such by an
occurrence of< 1%, with genetics being the primary underlying factor in the majority of these …