December 2009
The Newsletter of ROPARD
Timely Clinical Management Yields Good Vision
in Retinopathy of Prematurity
Gene Therapy
Gene therapy first received a lot of
press in the late 1980s and early 90s
when viral gene transfer was being
investigated. Some early successes
were followed by very disappointing
results. The premature media-hype
added to the disappointment and
set gene therapy back a few years.
Thankfully, a few dedicated scientists
and clinicians quietly persisted in their
endeavor for successful gene transfer
of DNA as a therapy. We now have a
successful clinical trial treating patients
with an inherited disease of blindness
(Leber’s Congenital Amaurosis - LCA)
with gene replacement.
The basic description of gene therapy
is the stable transfer of genetic material
into a host cell. Although it sounds
simple it is a daunting task. While
some diseases are perfect targets for
gene therapy most are not. There are
three questions that must be addressed
before designing a gene therapy.
1. Is the disease causing gene known?
Surprisingly, most diseases are
not caused by a single gene defect
which limits the diseases that can
be targeted. For instance, Retinitis
Pigmentosa has over 9 genes that
may be involved, and a single gene
may have multiple mutations. The
most successful gene therapy will
address a disease that has the most
patients affected by a single genetic
mutation, such as Congenital
X-Linked Retinoschisis (the RS1
gene).
2. Is it possible to package the DNA
encoding the gene therapy? There
are a number of ways to introduce
the genetic material of interest
to the host, both viral and nonviral.
The viral vectors have been
utilized the most because of their
natural affinity for host cell invasion
and their ability to integrate the DNA
for long-term therapy. It is possible
to package the genetic therapy into a
viral shell and then use that virus’ own
mechanisms for entering a cell. The
non-viral options use either synthetic
capsules for transfer or cell-based
systems. Neither of these options,
however, allow for long-term stable
transfer of a gene.
Continued on page 2
SAVE THE DATE:
The Children’s Vision Award
and Visions of the Future
On May 8, 2010 ROPARD will honor
Dr. Jean Bennett and Dr. Albert
Maguire with the Children’s Vision
Award. Drs. Bennett and Maguire
have used gene therapy to reverse
near blindness. The award ceremony
and dinner will take place at the Rivera
Court in The Detroit Institute of Arts,
Detroit, Michigan.


ROPARD has available for parents, family and professionals
many items that are useful for the development of children
with retinopathy of prematurity and low vision.
DVD l:
Booklet:
Brochures:
“Management of Retinopathy of Prematurity and
Other Pediatric Related Diseases” $30
“Looking Ahead: A Parent’s Guide to the
Development of their Child with Retinopathy
of Prematurity” $10
“A Parent’s Guide to Their Premature
Infant’s Eyes” Pkg of 100--$25.
(available in English or Spanish)
Order at www.ROPARD.org; or call 1-800-788-2020.
Holiday Cards
Don’t forget to order your Holiday Cards!
Package of 12 for $15.
They are also available in Braille for $22.
Custom imprinting upon request.

THE ASSOCIATION FOR RETINOPATHY OF
PREMATURITY AND RELATED DISEASES
Board of Directors
John Baker, M.D.
Margaret Cooney Casey
Patrick J. Droste, M.D.
Philip Hessburg, M.D.
David S. Hooker
Leslie Hooker
Jeffrey Maisels, M.D.
Raymond Margherio, M.D.
(1939-2000)
Rita Margherio
Thomas McAskin
Kay White Meyer
Edward O’Malley, M.D.
Venkat Reddy, Ph.D.
Caron Trese
Lori Webb
George A. Williams, M.D.
Kimberly Williams
Paul R. Ziegler
Michael Trese, M.D.
Co-Medical Director
Antonio Capone, Jr., M.D.
Co-Medical Director
Susan Campbell
Administrative Director
Paula Korelitz
Outreach Director