Method for implementing the design of synthetic nucleic acid molecules for gene therapies in rare diseases

The invention relates to methods that may be used to identify critical codons and design synthetic nucleic acid molecules of disease-causing genes. Synthetic nucleic acid sequences may be designed from a reference nucleic acid sequence, for example, to optimize heterologous expression of the nucleic acid sequence in a particular tissue of a host organism. Alternatively, synthetic nucleic acid sequences may be designed de novo to encode a desired polypeptide. Such synthetic nucleic acid sequences may be used for example in gene therapy or other therapeutic applications.