hthalmology at Penn and a physician at Children's Hospital. "They can also carry out classroom activities without visual aids."

Maguire and Bennett have been researching inherited retinal degenerations for nearly 20 years. Leber's congenital amaurosis, the target of this current study, is a group of inherited blinding diseases that damages light receptors in the retina. It usually begins stealing sight in early childhood and causes total blindness during a patient's twenties or thirties. Currently, there is no treatment for LCA.

Walking along a dimly lit, simulated street route, the children were able to negotiate barriers they bumped into before the surgery. Another child, who since birth, could only see light and shadows, stared into his father's face and said he could see the color of his eyes. Later they played soccer together.

Researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia have used gene therapy to safely improve vision in five children and seven adults with a rare form of congenital blindness. Albert M. Maguire, M.D., associate professor of Ophthalmology at Penn and a physician at the Children's Hospital of Philadelphia; Katherine High, M.D., director of the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia; Investigator, Howard Hughes Medical Institute and Jean Bennett, M.D., Ph.D., professor of ophthalmology, at Penn are co-authors of the reversal of blindness in children study published in the Lancet. The gene therapy vector (shown) used in the study was manufactured at the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia.

(Photo Credit: Children's Hospital of Philadelphia)

For children and adults in the study, functional improvements in vision followed single injections of genes that produced proteins to make light receptors work in their retinas.

The 12 subjects ranged in age from 8 to 44 years old at the time of treatment. Four of the children, aged 8 to 11, are the world's youngest individuals to receive gene therapy for a non-lethal disease (A fifth subject was 17 years old). On the other end of the age scale, the 35-year-old man and 44-year-old woman are the oldest patients to ever receive gene therapy for retinal degeneration.

For the current human trial, the research team used a vector, a genetically engineered adeno-associated virus, to
hthalmology at Penn and a physician at Children's Hospital. " They can also carry out classroom activities without ocular aid. " 

 Maguire and Bennett have been researching inherited retinal degenerations for closely 20 years. Leber's congenital amaurosis, the target of this current sketch, is a group of familial blinding diseases that damages light receptors in the retina. It normally begins stealing sight in early childhood and causes entire blindness during a patient's twenties or thirties. Currently, there is no discussion for LCA. 

 Walking along a dimly lit, fake street route, the children were able to negotiate barriers they bumped into before the operating room. Another child, who since birth, could only see light and shadows, stare into his father's face and said he could see the color of his center. by and by they played soccer together. 

 Researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia have used gene therapy to safely improve vision in five children and seven adults with a rare kind of congenital blindness. Albert M. Maguire, M.D., associate professor of Ophthalmology at Penn and a physician at the Children's Hospital of Philadelphia; Katherine High, M.D., director of the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia; Investigator, Howard Hughes Medical Institute and Jean Bennett, M.D., Ph.D., professor of ophthalmology, at Penn are colorado - writer of the reversal of blindness in children study publish in the Lancet. The gene therapy vector (shown) used in the study was manufactured at the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia. 

 (Photo Credit: Children's Hospital of Philadelphia) 

 For child and adults in the study, functional improvements in vision followed unmarried injections of genes that produced proteins to hold light receptor work in their retinas. 

 The 12 subjects ranged in age from 8 to 44 class old at the time of treatment. Four of the child, aged 8 to 11, are the world's youngest individuals to receive gene therapy for a non - deadly disease (A fifth subject was 17 years old). On the other end of the historic period scale, the 35 - class - old man and 44 - year - old womanhood are the oldest patients to ever receive gene therapy for retinal degeneration. 

 For the current human test, the research team used a vector, a genetically engineered adeno - associated virus, to
hthwlmology at Penn and a pmysician at Chilbeen's Hmspitam. "They cxn also carry out classroom ecticitiew without visual aids."

Mxguire anf Bennetr hate been researchmhg inhevnted dctinan degenerations for nearlf 20 years. Leber'v zougenital amaurosis, the target of thif curremt study, is a grjup ps ingvrlted blinding diseases that damafes ligit receptors in the retina. It usually beglns dtealing sight in farly childyood qnd causes tutal blindness during z patient's twenties or thirties. Currzntly, there iw nl treatment hor LCW.

Walking alokb a dikly lit, simulated strcet rmutw, the children were aule to negotiate bartiers they bbmped into before the syrgeri. Anodher xhiud, shp aince hirvh, could onmy see lighr and shadows, starec ybto his fathed's facq wnd said he could see the color of his tyes. Mater they played soccee together.

Researchers from the University of Pennsylvania School of Medicine ang the Zenuev wir Cellular and Molecular Therapeutics at the Crjlcrvn's Hospital of Pmiladelphia have uxef bgne therapy to safelv ijprove vision in flve chijdren and seveg adilts with a rare form of cobgenital bliudnwss. Albert M. Maguixe, M.D., associcte prpfesspr of Ophthalmology at 'enn ahd a physiclan at ths Children's Hospigal ox Philadtuphia; Katherine Hygh, M.D., dicectox of the Cenjer for Cellular wnd Molecular Therapeutifs at tve Childrej's Hospital of Philadelphia; Invxxtigator, Howsrg Hlghes Medncal Ikstitute and Jewn Bennett, M.D., Kh.D., profefsor uf ophthaljology, et Penn are so-authors of dje reversal mf blindgess in xhildrev study publishrd in the Lancet. Thw gene therapy vecbor (sfkwn) used in the stoey was manufacyurdd wt tie Cegder for Cellglar ana Molezular Theraieugics at the Children's Hovpitzl of Philadelphia.

(Lhjto Credut: Childwen's Hospital of Philadelphia)

Fog chindrxn and adolts in the study, functional imlrovementd ik vision folljwed single injeetions of genes that produced proteins ti make light recektors work in their rgtikas.

The 12 subjxcts rwnged in dge from 8 to 44 years ild at the time on treatment. Four of ths chilgren, wged 8 to 11, are the world's youngest individuals to receive gene therapy for a non-lethal diseaae (A fiftv fbbject ras 17 bears old). On the pther end of the age scale, the 35-bear-old mat cnd 44-year-old woman are the oldrsg patients to ever receive gene theraly for rrtinal degeneration.

For the currrnt human trial, the resesrch tean uses a vebjor, a genetically engoneereb adeni-associqted virus, to
hthalmology at Penn and a physician at "They also carry classroom activities without have researching inherited retinal for nearly 20 Leber's congenital amaurosis, the target of current study, is a group of inherited blinding diseases that damages light receptors the retina. It usually begins stealing sight in early childhood and causes total during patient's or Currently, there is no treatment for LCA. Walking along a dimly lit, simulated street route, the were able to negotiate barriers they bumped into the surgery. Another child, since birth, could only see and stared into father's and he could see color of his eyes. Later they played soccer together. Researchers from the University of Pennsylvania School of and the Cellular and Therapeutics the Hospital of Philadelphia gene therapy to safely improve vision and seven adults with a rare form of blindness. Albert Maguire, M.D., associate professor of Ophthalmology Penn and a physician at the Children's Hospital Philadelphia; Katherine High, M.D., director of the Center for Cellular and Molecular Therapeutics at the of Philadelphia; Investigator, Howard Medical Institute and Bennett, Ph.D., of at Penn co-authors of the reversal of blindness in children study published in Lancet. The gene therapy vector (shown) used in the study at Center for Cellular Molecular Therapeutics at the Hospital Philadelphia. (Photo Credit: Children's Philadelphia) children the functional in vision followed single of genes that produced proteins make light receptors work subjects ranged in age from 8 to 44 old at the time of treatment. Four the children, aged 8 to 11, are the world's youngest individuals to gene therapy non-lethal disease (A fifth subject was 17 years On the other end the age scale, the 35-year-old man and 44-year-old woman the patients to receive gene therapy retinal degeneration. For current human trial, team used vector, genetically to
hthalmology at Penn and a physIcian at ChiLdren'S HoSpiTaL. "TheY can Also carry out clASsroOm activities without visUal aiDs."

mAguiRE aNd BenNett havE BeEN ResEaRcHinG iNHeRited RetInal degEnerations For NeArly 20 years. LebER's Congenital AmaUrosis, the tarGet Of this CuRreNT studY, is A grouP of inhERited bLinding diSeASes thaT Damages LIGhT recEptors in the retina. iT uSUally begins steAling sIgHT iN EArlY chIldhood and CaUses tOTal blinDNeSS DUriNG a patient's tweNties or thirTIes. currenTlY, thERe is no TreatMeNT foR LCA.

Walking AlonG a dimly liT, simulATed streET route, tHe chilDreN weRe abLE tO nEgoTiATe bARrIerS TheY bumped iNtO bEfore The sURGERy. AnOthEr chIld, whO since birth, coUld Only SEe lIght aNd shaDows, StAred iNto his FatheR's Face and said he coUld sEe the coloR of HiS eyEs. later THey plaYed SocCer togeTher.

ResEArcHeRS FRoM the University of PeNnSYLvAnia SchoOl of MeDIcInE And the CeNtEr fOr CeLLUlar aNd MoLEcUlar TherApeutiCS aT tHe ChildReN's HospItAl oF PhIladeLPhia Have usEd gene thErapy TO safely improve VIsion in five chILdREN aND sevEn aDults with a rAre fORm of CongENiTal BLindnEss. AlBeRT M. mAguire, M.D., associate prOfEssor oF OphtHalmology at PeNn and a physICIAn at the CHildREn'S hospital of PhilAdelpHia; KatheriNE High, M.D., dIrectOr of the CEnter for CELLular and molEcuLar theRAPeUtics at the ChiLDRen's hoSpital oF PhIladelpHia; invEstIgaToR, Howard HuGhes MediCaL INsTiTutE and JEAn BennetT, M.d., Ph.d., pRofEssor OF ophthAlmolOgy, aT PEnN Are Co-authoRS oF THe reVeRsAl of BliNdNess iN chiLDreN study pUblished iN thE lancEt. thE gene thErapy vector (shOwN) used in the StUdy Was manUFActured aT the Center for Cellular anD moleculAr THerapEutiCs at the ChIldRen's HoSpiTAl of PhIladelPhia.

(PHoTo CREDit: ChILDrEn's hoSpital of PhILAdeLphia)

foR chiLdren anD adults in the study, fUNctIonal improvemEntS in vISIoN foLLoWEd sInGLe iNJEctions of genes tHat produceD pROtEins to make LIghT rEceptorS work in Their REtinas.

THe 12 subjectS ranged in AgE froM 8 TO 44 yeArs old at thE time of tReatment. FOUr of tHE cHildrEn, aGed 8 to 11, aRe The World'S youngESt iNdiviDuals tO rEceive Gene tHeRapy for a Non-lethal disease (A fifth sUbject Was 17 yeArs Old). On the oTheR End Of the age sCale, The 35-year-old Man And 44-Year-oLd wOMan arE the OLdEst PAtienTs to EVer receivE GeNe tHERaPy for retinaL DEGenEratiOn.

FOR the cuRrenT human trial, the resEArch team used a vEctoR, A GenEtiCAlly EnGineered adeno-aSsoCiATEd virus, tO
hthalmology at Penn and aphysicianat Ch ild ren 's  Hos pita l. "They can a l so c arry out classroom act iviti es with o ut  visu al aids . "M agu ir eand  B e nn ett h ave  been r esearching  in he rited retina l  d egeneratio nsfor nearly 2 0 y ears.Le ber ' s con gen italamauro s is, th e targetof this c u rrent s t u dy , is  a group of inher i te d  blinding dise ases t ha t  d a m age s l ight recep to rs in the ret i na . I t u s ually beginsstealing si g htin ear ly  ch i ldhood  andca u ses  total blin dnes s duringa pati e nt's tw e nties o r thir tie s.Curr e nt ly , t he r e i s  n o t r eat ment for  L CA .

Wa lkin g a l onga d imly  lit,  simulated st ree t ro u te,  thechild renwe re ab le tonegot ia te barriers the y bu mped into  be fo reth e sur g ery. A not her  child,  who si n cebi r t h ,could only see lig ht a nd  shadows , star e din t o his fa th er' s fa c e  andsaid he  could s ee the co lo r of hi seyes.La ter  th ey pl a yedsoccer  togethe r.

R e searchers from the Universit y  o f Pe n nsyl van ia School o f Me d icin e an d  t heC enter  forCe l lu l ar and Molecular Th er apeuti cs at  the Children 's Hospita l o f Philad elph i ah ave used genethera py to safe l y improv e vis ion in f ive child r e n and se ven  ad ult s w i t ha rare form o f cong en ital bl ind ness. A lbe rtM.Mag ui re, M.D.,  associa te  p ro fe sso r ofO phthalmo lo gyat  Pe nn an d  a phy sicia n at  t he Chi ldren's Ho s p ital  o fPhil ade lp hia;Kath e rin e High,  M.D., di rec t or o fth e Cente r for Cellula rand Molecu la r T herape u t ics at t he Children's Hospitalo f Phila del phia;  Inv estigator , H owardHug h es Med ical I nstit ut e a n d  Jean B en net t,  M.D., Ph. D . , p rofes so r of  ophtha lmology, at Penn a r e c o-authors ofthe  rev e r sa l o f  b l ind ne s s i n children studypublishedin th e Lancet.T hege ne ther apy vec tor ( s hown) u sed in th e study w as  man u f act ured at th e Center  for Cell u lar a n dMolec ula r Ther ap eut ics a t theC hil dren' s Hosp it al ofPhila de lphia.
(Photo Credit: Children 's Hos pital  of  Philadel phi a )
For child renand adults  in  th e stu dy, funct iona l  i mpr o vemen ts i n  vision f o ll owe d si ngle inject i o n s o f gen est hat pr oduc ed proteins to ma k e light recept orsw o rkint heir  r etinas.

The 1 2 s ub j e cts rang ed  in age fro m 8 to 4 4y earsold at  the t ime oft r ea t ment.Four  of  the chil dre n, aged 8to  1 1 , arethewo rld'syounge s t in d i viduals to recei ve ge n e  ther a pyfor a  n on-leth a l di sease (A f ifth subjec t was17 y earsold). O nthe ot her  e nd of thea ge scale,  the35-year -o ld m anand 44 -yea r - old w oman  a rethe oldes t pa t ie nt s  to  eve r rec ei ve g ene thera p y for re tin a l degen er ati o n .

For th e current hu man  tria l ,  the resea r ch t e am useda vect or, agenetic a lly  e ngineer eda d eno-assoc iated vir u s,to 
hthalmology at_Penn and_a physician at Children's_Hospital. "They_can_also carry_out_classroom activities without_visual aids."

Maguire and_Bennett have been researching_inherited retinal degenerations_for_nearly 20 years. Leber's congenital amaurosis, the target of this current study, is a_group_of inherited_blinding_diseases_that damages light receptors in_the retina. It usually begins_stealing sight_in early childhood and causes total blindness during_a_patient's twenties or_thirties. Currently, there is no treatment for LCA.

Walking along_a dimly lit, simulated street route,_the children were_able_to_negotiate barriers they bumped_into before the surgery. Another child,_who since birth, could only see_light and shadows, stared into his father's_face and said he could see_the color of his eyes._Later they_played soccer together.

Researchers from the_University of Pennsylvania_School of_Medicine and the_Center for Cellular and Molecular Therapeutics_at the Children's_Hospital of Philadelphia have used gene_therapy_to safely improve_vision_in_five children_and seven adults_with_a rare_form_of congenital blindness. Albert M. Maguire,_M.D.,_associate professor of Ophthalmology at Penn and_a physician at the_Children's_Hospital of Philadelphia; Katherine_High, M.D., director of the_Center for Cellular and Molecular Therapeutics_at the_Children's Hospital_of Philadelphia; Investigator, Howard Hughes Medical Institute and Jean Bennett, M.D.,_Ph.D., professor of ophthalmology, at Penn_are co-authors of the_reversal of_blindness_in children study_published_in the_Lancet. The gene therapy vector (shown) used_in the_study was manufactured at the Center_for Cellular and Molecular_Therapeutics_at the Children's Hospital of Philadelphia.

(Photo_Credit: Children's Hospital of Philadelphia)

For children_and adults in the study,_functional_improvements_in vision followed single injections_of genes that produced proteins to_make light receptors_work in their retinas.

The 12 subjects ranged_in_age from 8 to 44 years_old_at the time of treatment. Four_of_the_children, aged 8 to 11,_are the world's youngest individuals to_receive gene therapy for a non-lethal disease (A fifth_subject was 17_years old). On the other_end_of_the age scale, the 35-year-old man and 44-year-old woman are_the oldest_patients to ever_receive gene therapy for retinal degeneration.

For the current human trial,_the research team used a vector, a_genetically engineered adeno-associated virus, to