Current Edition

Gene Therapy in Rare Diseases

Many rare diseases are “monogenic” – that is, they are the consequence
of a defect in a single gene. Daniel Mazzolenis and Jozsef Palatka at
INC Research/inVentiv Health discuss how initial attempts at gene
therapy for monogenetic diseases were unsuccessful both for safety
and efficacy reasons, but with current scientific advances in the field,
gene therapy can offer life-changing efficacy.