Which of the following is FALSE regarding Gaucher Disease?

Gaucher Disease is indeed characterized by several key features, and the assertion that it can be treated with gene therapy is currently not accurate. While gene therapy has shown promise in various genetic disorders, as of now, it is not an established treatment modality for Gaucher Disease.

The disorder is an autosomal recessive condition, meaning it requires two copies of the mutated gene for the disease to manifest. This genetic basis is well-established in medical literature.

The disease arises due to a deficiency of the enzyme glucocerebrosidase, which is crucial for the breakdown of glucocerebrosides. Its deficiency leads to the accumulation of glucosylceramide, particularly in macrophages, which play a significant role in immune response and can lead to various complications associated with the disease.

There are therapies available for managing Gaucher Disease, such as enzyme replacement therapy and substrate reduction therapy, which help to alleviate symptoms and reduce the accumulation of harmful substances. However, gene therapy, as a treatment option, has not yet been validated and used in clinical settings for Gaucher Disease.