A research paper published in Nature journal overnight revealed that scientists had successfully edited the genomes of human embryos to prevent a mutation that causes hypertrophic cardiomyopathy, an inherited condition that causes sudden heart failure in young people.
Dr Nathan Palpant, Group Leader, Genomics of Development and Disease Division and Co-Director, Queensland Facility for Advanced Genome Editing at IMB, comments on the development below.
"Acquired genetic diseases remain a significant cause of morbidity and mortality worldwide and provide significant rationale for in vitro fertilization and genetic testing of embryos," Dr Palpant said.
"In recent years, the development of genome editing technologies such as CRISPR-Cas9 has provided a powerful means to modify DNA.
"While researchers use this technology routinely for experiment work, the application of this approach in human embryos is a significant advance with implications for manipulating the genome of future generations.
"The publication by Ma et al. in Nature provides important new insights into the use of CRISPR-Cas9 for gene correction of disease-causing mutations.
"The findings reveal new mechanisms by which DNA repair occurs in the embryo and evidence for specificity of the genetic modification.
"These findings add to a growing field of studies showing the power of CRISPR-editing as a therapeutic for acquired and inherited diseases.
"The ethical challenges of defining the boundaries for genome editing in this field will require careful consideration as we increasingly gain the capacity to manipulate the human genome."
Read a full expert round-up at the Australian Science Media Centre website.
