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RE: ANT--- PS

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Oh, one further question. Do you know if the ANT testing was done on fresh

or frozen tissue? I know Cleveland does some testing on fresh and some

testing on frozen from each biopsy. I just wondered which was used for ANT.

Barbara

_____

From: [mailto: ] On Behalf

Of Joanne Kocourek

Sent: Tuesday, June 21, 2005 9:52 AM

To:

Subject: RE: ANT

Dr. Hoppel (Cleveland CIDDM) did the ANT diagnosis. He was so surprised

that he actually has rechecked the study at least three times. It was been

a positive study each time. Dr. Wallace is the ANT expert in the US. So

given the girls also have identified defects in Complex I, III and IV, I

decided to discuss this atypical mix with him at the conference. (He was

very approachable). He said that, from his perspective, this mix is unusual

and is likely the result of severely abnormal dysfunction both within the

mitochondria and across the inner mitochondrial membrane. He is positive

that in my girls their base defect lies within mtDNA. Dr. Shoffner is doing

an evaluation of their entire mitochondrial genone and, to date, has not

identified any " known " mutations or deletions, etc. One curious

comment/questions he posed was, " Your daughter's are significantly

affected, aren't they? " How did he know??? Funny, they look " fine " but

medically are quite compromised with major organ

system involvement.

Barbara Seaman wheatchild@...> wrote:Joanne, Do you know if

there is any genetic connection between the two

defects your daughter has? In other words, is it possible that the ANT

defect is secondarily causing the deficiencies found in the respiratory

chain or are there two separate mutations, one for each defect? Or has this

even been defined? I ask because I also have two mitochondrial defects,

though different from your daughter's. You are the only other person I can

remember talking about two defects in one patient, so I am interested and

curious. Do you mind sharing who did the ANT defect diagnosis?

Thanks.

Barbara

_____

From: [mailto: ] On Behalf

Of Joanne Kocourek

Sent: Tuesday, June 21, 2005 9:11 AM

To:

Subject: Re: ANT

Adenine nucleotide translocators

General characteristics

Most abundant mitochondrial proteins

Integral components of inner mitochondrial membrane

Facilitate exchange of ADP and ATP between cytosol & mitochondria

ohgminion rakshasis@...> wrote:

Sorry, what is ANT?

Thanks,

RH

Joanne Kocourek (mom to , lies, and )

visit us at: http://www.caringbridge.org/il/annakris

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Oh, this is most interesting, as I also have an FOD. I will pass on

something about Dr. Wallace's work to my doctors and see how they respond.

Thanks again. Much appreciated.

Barbara

_____

From: [mailto: ] On Behalf

Of Joanne Kocourek

Sent: Tuesday, June 21, 2005 10:31 AM

To:

Subject: RE: ANT--- PS

I believe that the ANT studies are performed after the tissue has been

frozen (given Dr. Hoppel repeated the studies over a year later, some of the

study was definitely done using frozen tissue). The RTC studies are

completed when the sample is fresh.

As for ANT genetics....

increased expression of ANT-1 and ATP synthase: subunit mRNAs have been

described in patients with respiratory defects resulting from mtDNA point

mutations and increased mRNAs encoding cytochrome oxidase (COX IV and COX

VI) were also observed. While eukaryotic cells can respond to mitochondrial

dysfunction by a modulation of nuclear gene expression, evidence for

signaling pathways and transcriptional mechanisms are lacking in most

instances. Several studies have also questioned mt tRNA abnormalities. I

honestly believe that there can be multiple genetic reasons for a particluar

abnormality or mix of abnormalities. The science is just too young for a

complete body of knowledge. We are positive mtDNA is responsible in my

girls we just don't know where/how. Dr. Wallace recently identified a novel

homogygous mt related defect for another family (with an ANT defect and

FOD), so his work is continuing.

Barbara Seaman wheatchild@...> wrote:

Oh, one further question. Do you know if the ANT testing was done on fresh

or frozen tissue?

Joanne Kocourek (mom to , lies, and )

visit us at: http://www.caringbridge.org/il/annakris

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If you don't mind my asking, which FOD? My girls seem to have a VLCFAD (testing

still in process). Dr. Cohen is also highly suspicious of GAII.

Barbara Seaman wheatchild@...> wrote:Oh, this is most interesting,

as I also have an FOD. I will pass on

something about Dr. Wallace's work to my doctors and see how they respond.

Thanks again. Much appreciated.

Barbara

_____

From: [mailto: ] On Behalf

Of Joanne Kocourek

Sent: Tuesday, June 21, 2005 10:31 AM

To:

Subject: RE: ANT--- PS

I believe that the ANT studies are performed after the tissue has been

frozen (given Dr. Hoppel repeated the studies over a year later, some of the

study was definitely done using frozen tissue). The RTC studies are

completed when the sample is fresh.

As for ANT genetics....

increased expression of ANT-1 and ATP synthase: subunit mRNAs have been

described in patients with respiratory defects resulting from mtDNA point

mutations and increased mRNAs encoding cytochrome oxidase (COX IV and COX

VI) were also observed. While eukaryotic cells can respond to mitochondrial

dysfunction by a modulation of nuclear gene expression, evidence for

signaling pathways and transcriptional mechanisms are lacking in most

instances. Several studies have also questioned mt tRNA abnormalities. I

honestly believe that there can be multiple genetic reasons for a particluar

abnormality or mix of abnormalities. The science is just too young for a

complete body of knowledge. We are positive mtDNA is responsible in my

girls we just don't know where/how. Dr. Wallace recently identified a novel

homogygous mt related defect for another family (with an ANT defect and

FOD), so his work is continuing.

Barbara Seaman wheatchild@...> wrote:

Oh, one further question. Do you know if the ANT testing was done on fresh

or frozen tissue?

Joanne Kocourek (mom to , lies, and )

visit us at: http://www.caringbridge.org/il/annakris

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Are your daughters by any chance riboflavin-responsive? I don't know for sure,

but I doubt it. They take relatively high doses of riboflavin and

signs/symptoms have not lessened.

GA II is a relatively new " physician thought " for them. Dr. Cohen suggested it

a few months ago. He ruled out all of the leukodystrophies.

Children's Hospital was trying to run more formal fibrooblast studies. What

they completed showed a variety of abnormalities and then they ran out of cells.

They are waiting for more cells from Mayo and will then redo all of the studies.

Their Complex I, II and IV function ranges from 0-40% depending on the study.

TO date only the isolated novel point mutation has been found and Dr. Shoffner

didn't believe it was the cause of teh problem.

Information

regarding ANT defects is quite limited. Some of the articles are specific to

ANT alone and others discuss more global implications/deficiencies.

Dr. Wallace is the ANT guru! He is at UC Irvine (relocated from Emory).

Joanne Kocourek (mom to , lies, and )

visit us at: http://www.caringbridge.org/il/annakris

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Thanks!

B

_____

From: [mailto: ] On Behalf

Of Joanne Kocourek

Sent: Tuesday, June 21, 2005 11:45 AM

To:

Subject: RE: ANT--- PS

Are your daughters by any chance riboflavin-responsive? I don't know for

sure, but I doubt it. They take relatively high doses of riboflavin and

signs/symptoms have not lessened.

GA II is a relatively new " physician thought " for them. Dr. Cohen suggested

it a few months ago. He ruled out all of the leukodystrophies.

Children's Hospital was trying to run more formal fibrooblast studies. What

they completed showed a variety of abnormalities and then they ran out of

cells. They are waiting for more cells from Mayo and will then redo all of

the studies. Their Complex I, II and IV function ranges from 0-40%

depending on the study. TO date only the isolated novel point mutation has

been found and Dr. Shoffner didn't believe it was the cause of teh problem.

Information regarding ANT defects is quite limited. Some of the articles

are specific to ANT alone and others discuss more global

implications/deficiencies.

Dr. Wallace is the ANT guru! He is at UC Irvine (relocated from

Emory).

Joanne Kocourek (mom to , lies, and )

visit us at: http://www.caringbridge.org/il/annakris

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Thanks!

B

_____

From: [mailto: ] On Behalf

Of Joanne Kocourek

Sent: Tuesday, June 21, 2005 11:45 AM

To:

Subject: RE: ANT--- PS

Are your daughters by any chance riboflavin-responsive? I don't know for

sure, but I doubt it. They take relatively high doses of riboflavin and

signs/symptoms have not lessened.

GA II is a relatively new " physician thought " for them. Dr. Cohen suggested

it a few months ago. He ruled out all of the leukodystrophies.

Children's Hospital was trying to run more formal fibrooblast studies. What

they completed showed a variety of abnormalities and then they ran out of

cells. They are waiting for more cells from Mayo and will then redo all of

the studies. Their Complex I, II and IV function ranges from 0-40%

depending on the study. TO date only the isolated novel point mutation has

been found and Dr. Shoffner didn't believe it was the cause of teh problem.

Information regarding ANT defects is quite limited. Some of the articles

are specific to ANT alone and others discuss more global

implications/deficiencies.

Dr. Wallace is the ANT guru! He is at UC Irvine (relocated from

Emory).

Joanne Kocourek (mom to , lies, and )

visit us at: http://www.caringbridge.org/il/annakris

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Joanne

Do you know what gene was found that was homogygous. I have one at

A3796G (ACC.GCC, thr.Ala,N in Complex I. Dr. Shoffner thought that

since my mother also had the same defect and it was homogenious, that

it was insignificant.

laurie

> I believe that the ANT studies are performed after the tissue has been frozen

(given Dr. Hoppel repeated the studies over a year later, some of the study was

definitely done using frozen tissue). The RTC studies are completed when the

sample is fresh.

>

> As for ANT genetics....

> increased expression of ANT-1 and ATP synthase: subunit mRNAs have been

described in patients with respiratory defects resulting from mtDNA point

mutations and increased mRNAs encoding cytochrome oxidase (COX IV and COX VI)

were also observed. While eukaryotic cells can respond to mitochondrial

dysfunction by a modulation of nuclear gene expression, evidence for signaling

pathways and transcriptional mechanisms are lacking in most instances. Several

studies have also questioned mt tRNA abnormalities. I honestly believe that

there can be multiple genetic reasons for a particluar abnormality or mix of

abnormalities. The science is just too young for a complete body of knowledge.

We are positive mtDNA is responsible in my girls we just don't know where/how.

Dr. Wallace recently identified a novel homogygous mt related defect for another

family (with an ANT defect and FOD), so his work is continuing.

>

> Barbara Seaman wheatchild@...> wrote:

> Oh, one further question. Do you know if the ANT testing was done on fresh

> or frozen tissue?

>

> Joanne Kocourek (mom to , lies, and )

> visit us at: http://www.caringbridge.org/il/annakris

>

>

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Funny you should ask....it was definitely on an A-G

combination. I can't remember which number and every

other physician has tried to obtain a copy of the

report (and I've tried to obtain a replacement copy)

unsuccessfully. Our house sustaioned major water

damage while we were gone several years ago and I lost

alot of the paper files I had organized.

--- Laurie Fitzgerald laurie.fitzgerald@...>

wrote:

> Joanne

>

> Do you know what gene was found that was homogygous.

> I have one at

> A3796G (ACC.GCC, thr.Ala,N in Complex I. Dr.

> Shoffner thought that

> since my mother also had the same defect and it was

> homogenious, that

> it was insignificant.

>

> laurie

>

> On 6/21/05, Joanne Kocourek servedogmom@...>

> wrote:

> > I believe that the ANT studies are performed after

> the tissue has been frozen (given Dr. Hoppel

> repeated the studies over a year later, some of the

> study was definitely done using frozen tissue). The

> RTC studies are completed when the sample is fresh.

> >

> > As for ANT genetics....

> > increased expression of ANT-1 and ATP synthase:

> subunit mRNAs have been described in patients with

> respiratory defects resulting from mtDNA point

> mutations and increased mRNAs encoding cytochrome

> oxidase (COX IV and COX VI) were also observed.

> While eukaryotic cells can respond to mitochondrial

> dysfunction by a modulation of nuclear gene

> expression, evidence for signaling pathways and

> transcriptional mechanisms are lacking in most

> instances. Several studies have also questioned mt

> tRNA abnormalities. I honestly believe that there

> can be multiple genetic reasons for a particluar

> abnormality or mix of abnormalities. The science is

> just too young for a complete body of knowledge. We

> are positive mtDNA is responsible in my girls we

> just don't know where/how. Dr. Wallace recently

> identified a novel homogygous mt related defect for

> another family (with an ANT defect and FOD), so his

> work is continuing.

> >

> > Barbara Seaman wheatchild@...>

> wrote:

> > Oh, one further question. Do you know if the ANT

> testing was done on fresh

> > or frozen tissue?

> >

> > Joanne Kocourek (mom to , lies, and

> )

> > visit us at:

> http://www.caringbridge.org/il/annakris

> >

> > [Non-text portions of this message have been

> removed]

> >

> >

> >

> > Medical advice, information, opinions, data and

> statements contained herein are not necessarily

> those of the list moderators. The author of this e

> mail is entirely responsible for its content. List

> members are reminded of their responsibility to

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> with their physicians regarding changes in their own

> treatment.

> >

> > Personal attacks are not permitted on the list and

> anyone who sends one is automatically moderated or

> removed depending on the severity of the attack.

> >

> >

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