Gene Therapy for Dobermans with Dilated Cardiomyopathy

Dr. Meg Sleeper
Dr. Meg Sleeper with Scarlett and Ava, Dobermans and clients of the UF Small Animal Hospital, owned by Jaylee Padilla. (Photo by Sarah Carey)

The cardiology service at the University of Florida is looking to assess efficacy of therapeutic gene delivery using a virus vector.  The study is open label, so all dogs will be treated. To qualify for this study, the following criteria must be met:

Enrollment Status: Currently Enrolling

  • Inclusion Criteria:
    • Must be a Doberman
    • A diagnosis of occult stage dilated cardiomyopathy prior to onset of congestive heart failure which has been controlled with medical management
    • A circulating neutralizing antibody titer to the virus vector of less than 1:20
    • Clear of extra-cardiac disease, congenital heart disease or primary mitral valvular disease
    • Owner agreement to authorize participation with informed consent and collection of the heart following euthanasia or death from any cause.
  • Additional Information: 

    Eligibility for enrollment criteria: Procedures covered by the study include bloodwork, ECH, and ECG. Enrolled patients also receive repeat screenings at no cost. The value of the study procedure is estimated at $5,000. The cardiology exam ($200) is NOT covered by the  study, nor is the cost of thoracic radiographs if not provided by referring veterinarian.

  • Contact:  Contact the Cardiology Service at the Small Animal Hospital 352-392-2235.  If you have further questions or want to see if your dog qualifies, please complete the Cardiology Study Interest Form.
  • PI: Dr. Meg Sleeper, VMD, DACVIM (cardiology). Prior to setting an appointment, you are STRONGLY encouraged to email Dr. Sleeper that she can help pre-screen potential candidates and to clarify any fees you might incur.

Study Details:

At enrollment, an antibody titer, hematology and biochemistry panel will be used for screening purposes. Dogs will undergo a 3-minute electrocardiogram (ECG), a complete echocardiogram (ECHO) and the owners will complete a quality of life questionnaire. In order to participate in the study, dogs fulfilling the requirements will receive the gene therapy (cardiac injection with the virus vector: AAV2/6-ARC-s100a1). Standard medical management for DCM, and congestive heart failure, if applicable, will continue throughout the study in all dogs (pimobendan, angiotensin converting enzyme inhibitor and diuretic therapy). Owners will need to commit to re-evaluations at 2, 4, 6, 9, and 12 months at the University of Florida following therapy.

Funding for the research is provided through the efforts and generosity of the AKC Canine Health Foundation.

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