Gene Therapies Unlock New Possibilities for Curing Genetic Diseases
- Targeted treatment that corrects genetic mutations at the source
- Successful treatment of inherited retinal diseases, hemophilia, and spinal muscular atrophy
- Potential to provide lifelong cures for patients with single-gene disorders
Gene therapies available through Clinicians International's network are performed in compliance with the regulatory standards of the host country. These therapies may not be FDA-approved for all indications. It is essential for patients to have detailed discussions with their healthcare provider to understand the implications, risks, and benefits of gene therapies. Clinicians International collaborates with internationally recognized clinics to provide safe and effective treatments.
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has restored functional vision in patients with inherited retinal disease caused by mutations in the RPE65 gene, with sustained improvements observed over several years.
has shown remarkable efficacy in treating spinal muscular atrophy (SMA), with treated infants achieving developmental milestones such as sitting without support, which are rarely seen in untreated patients.Are you a good candidate for Gene Therapies?
Start your Free Pre-Consultation NowSpecific genetic
mutations
Patients with specific genetic mutations that are directly targeted by available gene therapies, such as mutations in the RPE65 gene (inherited retinal disease) or the SMN1 gene (spinal muscular atrophy), are ideal candidates.
Early diagnosis
and treatment
Patients diagnosed and treated early in the course of their disease tend to have better outcomes with gene therapy, as early intervention can prevent the progression of debilitating symptoms.
Absence of severe
immune response
Patients without a history of severe immune responses or allergies to viral vectors used in gene therapies are better candidates, as they are less likely to experience adverse reactions.
Prelimenary steps before
starting gene therapies
24/7 access to free pre-consultation
Through Clinicians International, you can take a thorough pre-consultation and get matched with the world’s best gene therapy specialists to help plan the rest of your life pain-free.
How it WorksFrequently asked Questions about gene therapies
Hear it from research papers
Four Success Stories:
in Gene Therapy - 2020This paper highlights four successful cases in gene therapy, including treatments for sickle cell disease, beta thalassemia, and spinal muscular atrophy, using CRISPR-Cas9 and other gene-editing technologies.
Gene Therapy for Cystic Fibrosis:
Progress and Challenges of Genome Editing - 2020This review discusses advancements in gene therapy for cystic fibrosis, focusing on genome editing techniques like CRISPR-Cas9 and their potential to correct genetic defects in the CFTR gene.
The Promise and the Hope:
of Gene Therapy - 2021This paper reviews the development and clinical success of gene therapies such as Zynteglo for beta thalassemia, discussing the progress in HSC gene therapy and its impact on genetic diseases.